[{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prevail Therapeutics Provides PR001 Program Update","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Not Applicable","leadProduct":"PR001","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Prevail Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Prevail Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Prevail Th.."},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Not Applicable","leadProduct":"GTX-102","moa":"UBE3A-AS expression","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"GeneTx Biotherapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"GeneTx Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"GeneTx Bio.."},{"orgOrder":0,"company":"Emerald Health Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Emerald Health Pharmaceuticals Granted Orphan Designation in Europe for Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Not Applicable","leadProduct":"EHP-102","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Emerald Health Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Oral","sponsorNew":"Emerald Health Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Emerald He.."},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Canada Pension Plan Investment Board ","pharmaFlowCategory":"D","amount":"$80.3 million","upfrontCash":"Undisclosed","newsHeadline":"Sigilon Therapeutics Announces $80.3 Million Series B Financing to Advance","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"March 2020","year":"2020","type":"Series B Financing","leadProduct":"SIG-001","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Sigilon Therapeutics","amount2":0.080000000000000002,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Sigilon Therapeutics \/ Canada Pension Plan Investment Board ","highestDevelopmentStatusID":"5","companyTruncated":"Sigilon Th.."},{"orgOrder":0,"company":"DiNAQOR AG","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","year":"2020","type":"Licensing Agreement","leadProduct":"DiNA-001","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DiNAQOR AG","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"DiNAQOR AG \/ BioMarin Pharmaceutical ","highestDevelopmentStatusID":"5","companyTruncated":"DiNAQOR AG.."},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme and GACI Global Initiate Study to Understand Severity and Impact of Disease in Patients with ENPP1 Deficiency and ABCC6 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","year":"2020","type":"Not Applicable","leadProduct":"INZ-701","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Inozyme Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Inozyme Ph.."},{"orgOrder":0,"company":"AmideBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Grants Orphan Drug Designation to Amidebio's Glucagon Analog for the Treatment of Congenital Hyperinsulinism","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","year":"2020","type":"Not Applicable","leadProduct":"AB-G023","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"AmideBio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Solution","sponsorNew":"AmideBio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"AmideBio \/.."},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LogicBio Therapeutics Announces Submission of Investigational New Drug Application (IND) for LB-001 for Methylmalonic Acidemia (MMA) and Highlights Key Corporate Milestones","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Not Applicable","leadProduct":"LB-001","moa":"MUt transgene","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"LogicBio Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"LogicBio Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"LogicBio T.."},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"PBM Capital","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"Taysha Gene Therapies Announce $30 Million Seed Financing and Strategic Partnership with the UT Southwestern Gene Therapy Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","year":"2020","type":"Financing","leadProduct":"TGTX-101","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Taysha Gene Therapies","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ PBM Capital","highestDevelopmentStatusID":"5","companyTruncated":"Taysha Gen.."},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Magenta Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Magenta's Technology Attracts Another Gene Therapy Developer Beam Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","year":"2020","type":"Collaboration","leadProduct":"MGTA-117","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Beam Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Beam Therapeutics \/ Magenta Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"Beam Thera.."},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to Neurogene\u2019s Gene Therapy for the Treatment of CLN5 Batten Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","year":"2020","type":"Not Applicable","leadProduct":"AAV-based engineered transgene","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neurogene","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene .."},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Inozyme Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Inozyme Pharma Acquires ENPP1 Deficiency Program Assets from Alexion Pharmaceuticals","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","year":"2020","type":"Acquisition","leadProduct":"INZ-701","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Alexion Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Alexion Pharmaceuticals \/ Inozyme Pharma","highestDevelopmentStatusID":"5","companyTruncated":"Alexion Ph.."},{"orgOrder":0,"company":"Neurogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurogene Appoints Kenneth Huttner, M.D., Ph.D., as Senior Vice President of Clinical Development","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","year":"2020","type":"Not Applicable","leadProduct":"AAV-based gene therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neurogene","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Neurogene \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene .."},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Taysha Gene Therapies","pharmaFlowCategory":"D","amount":"$63.0 million","upfrontCash":"$3.0 million","newsHeadline":"Abeona Therapeutics and Taysha Gene Therapies Enter into Licensing and Inventory Purchase Agreements for ABO-202","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","year":"2020","type":"Licensing Agreement","leadProduct":"ABO-202","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Abeona Therapeutics","amount2":0.059999999999999998,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Abeona Therapeutics \/ Taysha Gene Therapies","highestDevelopmentStatusID":"5","companyTruncated":"Abeona The.."},{"orgOrder":0,"company":"AmideBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Grants Rare Pediatric Disease Designation to Amide Bio's Glucagon Analog for the Treatment of Congenital Hyperinsulinism","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2020","year":"2020","type":"Not Applicable","leadProduct":"AB-G023","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"AmideBio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Solution","sponsorNew":"AmideBio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"AmideBio \/.."},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Taysha Gene Therapies","pharmaFlowCategory":"D","amount":"$63.0 million","upfrontCash":"$7.0 million","newsHeadline":"Abeona Therapeutics and Taysha Gene Therapies Enter into Licensing and Inventory Purchase Agreements for ABO-202","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","year":"2020","type":"Licensing Agreement","leadProduct":"ABO-202","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Abeona Therapeutics","amount2":0.059999999999999998,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Abeona Therapeutics \/ Taysha Gene Therapies","highestDevelopmentStatusID":"5","companyTruncated":"Abeona The.."},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Versant Ventures","pharmaFlowCategory":"D","amount":"$45.0 million","upfrontCash":"Undisclosed","newsHeadline":"Graphite Bio Launches with $45 Million Series A Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"September 2020","year":"2020","type":"Series A Financing","leadProduct":"Gene therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Graphite Bio","amount2":0.050000000000000003,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.050000000000000003,"dosageForm":"","sponsorNew":"Graphite Bio \/ Versant Ventures","highestDevelopmentStatusID":"5","companyTruncated":"Graphite B.."},{"orgOrder":0,"company":"University of North Carolina","sponsor":"Geneventiv Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Geneventiv Therapeutics Licenses Gene Therapy for Hemophilia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","year":"2020","type":"Licensing Agreement","leadProduct":"GENV HEM","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"University of North Carolina","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"University of North Carolina \/ Geneventiv Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"University.."},{"orgOrder":0,"company":"Allomek","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants AlloMek Orphan Designation for CIP-137401, Treatment of Neurofibromatosis Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","year":"2020","type":"Not Applicable","leadProduct":"CIP-137401","moa":"MEK kinase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Allomek","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Allomek \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Allomek \/ .."},{"orgOrder":0,"company":"WuXi Biologics","sponsor":"ViGeneron","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"ViGeneron and WuXi Advanced Therapies Enter Strategic Manufacturing Partnership For Next-Generation Ophthalmic Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","year":"2020","type":"Partnership","leadProduct":"VG901","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"WuXi Biologics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"WuXi Biologics \/ ViGeneron","highestDevelopmentStatusID":"5","companyTruncated":"WuXi Biolo.."},{"orgOrder":0,"company":"Neurogene","sponsor":"EcoR1 Capital","pharmaFlowCategory":"D","amount":"$115.0 million","upfrontCash":"Undisclosed","newsHeadline":"Neurogene Closes $115 Million Series B Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"December 2020","year":"2020","type":"Series B Financing","leadProduct":"AAV-based gene therapy","moa":"CLN5","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neurogene","amount2":0.12,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.12,"dosageForm":"","sponsorNew":"Neurogene \/ EcoR1 Capital","highestDevelopmentStatusID":"5","companyTruncated":"Neurogene .."},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sigilon Therapeutics Receives Orphan Drug Designation for SIG-005 for the Treatment of Mucopolysaccharidosis Type I","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","year":"2020","type":"Not Applicable","leadProduct":"SIG-005","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Sigilon Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Sigilon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Sigilon Th.."},{"orgOrder":0,"company":"French National Institute of Health and Medical Research","sponsor":"Tonix Pharmaceuticals Holding Corp","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Tonix Pharma Licenses Technology for Treating Prader-Willi Syndrome from the French National Institute of Health and Medical Research","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Peptide","productStatus":"Approved","date":"February 2021","year":"2021","type":"Licensing Agreement","leadProduct":"Oxytocin","moa":"Oxytocin receptor","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"French National Institute of Health and Medical Research","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"intranasal","sponsorNew":"French National Institute of Health and Medical Research \/ Tonix Pharmaceuticals","highestDevelopmentStatusID":"5","companyTruncated":"French Nat.."},{"orgOrder":0,"company":"Chinook Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chinook Receives Rare Pediatric Disease Designation from U.S. FDA for CHK-336 for Treatment of Primary Hyperoxaluria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","year":"2021","type":"Not Applicable","leadProduct":"CHK-336","moa":"LDH-A","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Chinook Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Oral","sponsorNew":"Chinook Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Chinook Th.."},{"orgOrder":0,"company":"Rescindo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Rescindo\u2019s Treatment for Kabuki Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","year":"2021","type":"Not Applicable","leadProduct":"RSC-57","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Rescindo Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Rescindo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Rescindo T.."},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PYC Therapeutics Highlights Progress in Its Transformation to a U.S. Clinical-Stage Company and 2021 Corporate Objectives","therapeuticArea":"Ophthalmology","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","year":"2021","type":"Not Applicable","leadProduct":"VP-001","moa":"PRPF31","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"PYC Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"PYC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"PYC Therap.."},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX701 for the Treatment of Wilson Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","year":"2021","type":"Not Applicable","leadProduct":"UX701","moa":"ATP7B copper transport","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Ultragenyx Pharmaceutical","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous infusion","sponsorNew":"Ultragenyx Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Ultragenyx.."},{"orgOrder":0,"company":"DTx Pharma","sponsor":"RA Capital Management","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"DTx Pharma Completes $100M Series B Financing to Advance its FALCON\u2122 Platform and Pipeline of RNA Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2021","year":"2021","type":"Series B Financing","leadProduct":"RNA-based therapeutics","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DTx Pharma","amount2":0.10000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.10000000000000001,"dosageForm":"","sponsorNew":"DTx Pharma \/ RA Capital Management","highestDevelopmentStatusID":"5","companyTruncated":"DTx Pharma.."},{"orgOrder":0,"company":"Adverum Biotechnologies","sponsor":"Lexeo Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"LEXEO Therapeutics Announces License Agreement and Consolidation of Pre-clinical Data Package","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","year":"2021","type":"Licensing Agreement","leadProduct":"LX2006","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Adverum Biotechnologies","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Adverum Biotechnologies \/ LEXEO Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"Adverum Bi.."},{"orgOrder":0,"company":"PYC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PYC's Lead Investigational Drug, VP-001, Demonstrates Another Key Functional Improvement in Patient-Derived Models","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2021","year":"2021","type":"Not Applicable","leadProduct":"VP-001","moa":"PRPF31","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"PYC Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"PYC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"PYC Therap.."},{"orgOrder":0,"company":"Agtc","sponsor":"SparingVision","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AGTC licenses Promoter Technology to SparingVision SAS","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","year":"2021","type":"Licensing Agreement","leadProduct":"SPVN06","moa":"RdCVF\/RdCVFL","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Agtc","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ SparingVision","highestDevelopmentStatusID":"5","companyTruncated":"Agtc \/ Spa.."},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme Pharma Publishes Data Supporting INZ-701 as a Potential Treatment for ENPP1 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","year":"2021","type":"Not Applicable","leadProduct":"INZ-701","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Inozyme Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Inozyme Ph.."},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme Pharma Presents Data from Burden of Illness Study in Patients with ENPP1 Deficiency and ABCC6 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","year":"2021","type":"Not Applicable","leadProduct":"INZ-701","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Inozyme Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Inozyme Pharma \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Inozyme Ph.."},{"orgOrder":0,"company":"Enzyvant","sponsor":"Aceragen","pharmaFlowCategory":"D","amount":"$226.0 million","upfrontCash":"Undisclosed","newsHeadline":"Aceragen Launches with Acquisition of Enzyvant\u2018s Investigational Therapy for Farber Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","year":"2021","type":"Acquisition","leadProduct":"Recombinant human acid ceramidase","moa":"Acid ceramidase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Enzyvant","amount2":0.23000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.23000000000000001,"dosageForm":"","sponsorNew":"Enzyvant \/ Aceragen","highestDevelopmentStatusID":"5","companyTruncated":"Enzyvant \/.."},{"orgOrder":0,"company":"SpliSense","sponsor":"Biotel Limited","pharmaFlowCategory":"D","amount":"$28.5 million","upfrontCash":"Undisclosed","newsHeadline":"SpliSense Secures $28.5 Million in Series B Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","year":"2021","type":"Series B Financing","leadProduct":"SPL84-23","moa":"CFTR","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"SpliSense","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"Inhalation","sponsorNew":"SpliSense \/ Biotel Limited","highestDevelopmentStatusID":"5","companyTruncated":"SpliSense .."},{"orgOrder":0,"company":"Vital Therapeutics & Formulations Pvt Ltd","sponsor":"Cambrian Biopharma","pharmaFlowCategory":"D","amount":"$32.0 million","upfrontCash":"Undisclosed","newsHeadline":"Vita Therapeutics Raises $32 Million in Oversubscribed Series A Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","year":"2021","type":"Series A Financing","leadProduct":"VTA-100","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vital Therapeutics & Formulations Pvt Ltd","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Vital Therapeutics & Formulations Pvt Ltd \/ Cambrian Biopharma","highestDevelopmentStatusID":"5","companyTruncated":"Vital Ther.."},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sigilon Preclinical Data International Symposium on MPS and Related Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2021","year":"2021","type":"Not Applicable","leadProduct":"SIG-005","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Sigilon Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Sigilon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Sigilon Th.."},{"orgOrder":0,"company":"PepGen","sponsor":"RA Capital Management","pharmaFlowCategory":"D","amount":"$112.5 million","upfrontCash":"Undisclosed","newsHeadline":"PepGen Announces Closing of $112.5 Million Crossover Financing to Advance Transformative Therapies for Neuromuscular Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2021","year":"2021","type":"Financing","leadProduct":"EDO51","moa":"Exon 51 inclusion","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"PepGen","amount2":0.11,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.11,"dosageForm":"","sponsorNew":"PepGen \/ RA Capital Management","highestDevelopmentStatusID":"5","companyTruncated":"PepGen \/ R.."},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"WuXi Biologics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Pharmaceuticals CAN103 Investigational New Drug Application Accepted by China\u2019s National Medical Products Administration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2021","year":"2021","type":"Not Applicable","leadProduct":"CAN103","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"CANbridge Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"CANbridge Pharmaceuticals \/ WuXi Biologics","highestDevelopmentStatusID":"5","companyTruncated":"CANbridge .."},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"US FDA Grants Orphan Drug Designation to Polaryx Therapeutics\u2019 PLX-200 to Treat GM2 Gangliosidoses","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"Approved","date":"August 2021","year":"2021","type":"Not Applicable","leadProduct":"Gemfibrozil","moa":"PPAR gamma","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Polaryx Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Polaryx Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Polaryx Th.."},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"SR One Capital Management","pharmaFlowCategory":"D","amount":"$75.0 million","upfrontCash":"Undisclosed","newsHeadline":"ADARx Raises $75 Million to Advance Growing Pipeline","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"Undisclosed","date":"September 2021","year":"2021","type":"Series B Financing","leadProduct":"Oligonucleotide-based Therapeutics","moa":"RNA","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ADARx Pharmaceuticals","amount2":0.080000000000000002,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"ADARx Pharmaceuticals \/ SR One Capital Management","highestDevelopmentStatusID":"5","companyTruncated":"ADARx Phar.."},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"iECURE","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Precision BioSciences and iECURE Announce License and Collaboration Agreement to Develop ARCUS-Based Gene Editing Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","year":"2021","type":"Collaboration","leadProduct":"PBGENE-PCSK9","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Precision BioSciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Precision BioSciences \/ iECURE","highestDevelopmentStatusID":"5","companyTruncated":"Precision .."},{"orgOrder":0,"company":"NeuExcell therapeutics","sponsor":"Spark Therapeutics, Inc","pharmaFlowCategory":"D","amount":"$190.0 million","upfrontCash":"Undisclosed","newsHeadline":"NeuExcell Therapeutics and Spark Therapeutics Announce Research Collaboration Agreement to Develop a Novel Gene Therapy for Huntington's Dis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","year":"2021","type":"Collaboration","leadProduct":"AAV-based Gene Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"NeuExcell therapeutics","amount2":0.19,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.19,"dosageForm":"","sponsorNew":"NeuExcell therapeutics \/ Spark","highestDevelopmentStatusID":"5","companyTruncated":"NeuExcell .."},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen To Present Research on Enhanced Delivery Treatments for Neuromuscular Disease at Two Scientific Conferences in September","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"September 2021","year":"2021","type":"Not Applicable","leadProduct":"EDO51","moa":"Exon 51 inclusion","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"PepGen","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"PepGen \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"PepGen \/ N.."},{"orgOrder":0,"company":"Healx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Healx Receives Ind and Orphan Drug Designation for Fragile X Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"Approved","date":"October 2021","year":"2021","type":"Not Applicable","leadProduct":"HLX-0201","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Healx","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Healx \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Healx \/ No.."},{"orgOrder":0,"company":"ContraFect Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ContraFect to Present Data on the Potential of DLAs to Treat Pulmonary Infections in Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2021","year":"2021","type":"Not Applicable","leadProduct":"CF-370","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ContraFect Corporation","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"ContraFect Corporation \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"ContraFect.."},{"orgOrder":0,"company":"University of L'Aquila","sponsor":"SiSaf Ltd","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"SiSaf Exercises Its Option Under Licencing Agreement on Gene Therapy for Rare Bone Disease and Announces Progress on Ind Enabling Studies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"ITALY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","year":"2021","type":"Collaboration","leadProduct":"SIS-101-ADO","moa":"CLCN7 gene mutation","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"University of L'Aquila","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"University of L'Aquila \/ SiSaf Ltd","highestDevelopmentStatusID":"5","companyTruncated":"University.."},{"orgOrder":0,"company":"GeneVentiv Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneVentiv Receives Orphan Drug Designation (ODD) for GENV-HEM for the Treatment of Hemophilia A or B With or Without Inhibitors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","year":"2021","type":"Not Applicable","leadProduct":"GENV HEM","moa":"Factor Va","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"GeneVentiv Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"GeneVentiv Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"GeneVentiv.."},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AVROBIO Receives Rare Pediatric Disease Designation from the U.S. FDA for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","year":"2021","type":"Not Applicable","leadProduct":"Lentiviral gene therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Avrobio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Avrobio \/ .."},{"orgOrder":0,"company":"ContraFect Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ContraFect\u2019s New Data Presented at NACFC Demonstrate Potential of Direct Lytic Agents to Treat Pulmonary Infections in Patients with Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","year":"2021","type":"Not Applicable","leadProduct":"CF-370","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ContraFect Corporation","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"ContraFect Corporation \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"ContraFect.."},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","amount":"$4.0 million","upfrontCash":"Undisclosed","newsHeadline":"Immusoft Receives $4M in Funding from the California Institute for Regenerative Medicine (CIRM)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"Approved","date":"November 2021","year":"2021","type":"Funding","leadProduct":"Iduronate Sulfatase","moa":"Enzyme Replacement","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Immusoft Corporation","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Immusoft Corporation \/ California Institute for Regenerative Medicine","highestDevelopmentStatusID":"5","companyTruncated":"Immusoft C.."},{"orgOrder":0,"company":"Amarna Therapeutics","sponsor":"C4 holding BV","pharmaFlowCategory":"D","amount":"$5.7 million","upfrontCash":"Undisclosed","newsHeadline":"Amarna Therapeutics Secures Additional \u20ac5 ($5.7) Million to Advance Its Transformative Gene Therapy Platform Towards Clinical Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","year":"2021","type":"Financing","leadProduct":"AMA005","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Amarna Therapeutics","amount2":0.01,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.01,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Amarna Therapeutics \/ C4 holding BV","highestDevelopmentStatusID":"5","companyTruncated":"Amarna The.."},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Beam Therapeutics Outlines Long-term Strategy for Base Editing Programs in Sickle Cell Disease and Provides Preclinical Data Updates at ASH","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","year":"2021","type":"Not Applicable","leadProduct":"BEAM-101","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Beam Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Beam Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Beam Thera.."},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"CMG-SDIC Capital","pharmaFlowCategory":"D","amount":"$60.0 million","upfrontCash":"Undisclosed","newsHeadline":"Neurophth Raised Over $60 Million USD in Series-C Financing to Advance Gene Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","year":"2021","type":"Series C Financing","leadProduct":"rAAV-ND4","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neurophth Therapeutics","amount2":0.059999999999999998,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.059999999999999998,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Neurophth Therapeutics \/ CMG-SDIC Capital","highestDevelopmentStatusID":"5","companyTruncated":"Neurophth .."},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Beam Therapeutics Reports Progress Across Ex Vivo and In Vivo Pipeline of Base Editing Therapeutics and Outlines Key Anticipated 2022 Milestones","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","year":"2022","type":"Not Applicable","leadProduct":"BEAM-101","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Beam Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Beam Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Beam Thera.."},{"orgOrder":0,"company":"Ractigen Therapeutics","sponsor":"SDIC Venture Capital","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ractigen Therapeutics Closes an Additional $30 Million in Series A+ Funding to Push Development of Its RNAa Platform","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","year":"2022","type":"Series A Financing","leadProduct":"RAG-06","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Ractigen Therapeutics","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Ractigen Therapeutics \/ SDIC Venture Capital","highestDevelopmentStatusID":"5","companyTruncated":"Ractigen T.."},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sigilon Therapeutics to Present Preclinical Data at the 18th Annual WORLDSymposium\u2122 on Lysosomal Diseases, MPS-1 and MPS-6","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","year":"2022","type":"Not Applicable","leadProduct":"SIG-005","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Sigilon Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Sigilon Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Sigilon Th.."},{"orgOrder":0,"company":"Juvena Therapeutics","sponsor":"California Institute of Regenerative Medicine","pharmaFlowCategory":"D","amount":"$3.9 million","upfrontCash":"Undisclosed","newsHeadline":"Juvena Therapeutics Awarded Highly Competitive CIRM Grant To Advance Lead Program, JUV-161, To Treat Myotonic Dystrophy Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","year":"2022","type":"Funding","leadProduct":"JUV-161","moa":"Myogenesis","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Juvena Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Juvena Therapeutics \/ California Institute of Regenerative Medicine","highestDevelopmentStatusID":"5","companyTruncated":"Juvena The.."},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen Announces Approval by Health Canada of CTA to Begin First in Human Trials of PGN-EDO51 to Treat Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2022","year":"2022","type":"Not Applicable","leadProduct":"EDO51","moa":"Exon 51 inclusion","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"PepGen","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"PepGen \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"PepGen \/ N.."},{"orgOrder":0,"company":"Opus Genetics","sponsor":"National Resilience","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Opus Enters Strategic Collaboration with Resilience for AAV-based Gene Therapy Development and Manufacturing for Inherited Retinal Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","year":"2022","type":"Collaboration","leadProduct":"OPGx-001","moa":"LCA5 gene","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Opus Genetics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Opus Genetics \/ National Resilience","highestDevelopmentStatusID":"5","companyTruncated":"Opus Genet.."},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Receives FDA Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Beta Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","year":"2022","type":"Not Applicable","leadProduct":"EDIT-301","moa":"CD34+ gene","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Editas Medicine","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Editas Medicine \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Editas Med.."},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Precision BioSciences to Present Preclinical In Vivo Gene Editing Research at Upcoming American Society of Gene & Cell Therapy 25th Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Not Applicable","leadProduct":"PBGENE-PH1","moa":"HO-1","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Precision BioSciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Precision BioSciences \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Precision .."},{"orgOrder":0,"company":"DepYmed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"DepYmed Receives FDA Rare Pediatric Disease and Orphan Drug Designations for Its Lead Clinical Candidate for the Treatment of Rett Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"PTP1B","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DepYmed","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"DepYmed \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"DepYmed \/ .."},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Opus Genetics Announces Promising New Data Highlighting Potential of AAV-based Gene Therapies for the Treatment of Rare Inherited Retinal Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Not Applicable","leadProduct":"OPGx-001","moa":"LCA5 gene","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Opus Genetics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Opus Genetics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Opus Genet.."},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Precision BioSciences Announces Preclinical Data Showcasing Premier In Vivo Gene Editing Capabilities at American Society of Gene & Cell Therapy Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Not Applicable","leadProduct":"PBGENE-PH1","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Precision BioSciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Precision BioSciences \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Precision .."},{"orgOrder":0,"company":"Neuronascent","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neuronascent Receives FDA Rare Pediatric Drug Designation for NNI-351 Treatment for Fragile X Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Not Applicable","leadProduct":"NNI-351","moa":"DYRK kinase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neuronascent","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Neuronascent \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Neuronasce.."},{"orgOrder":0,"company":"Passage Bio","sponsor":"University of Pennsylvania","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Passage Bio Receives FDA Clearance of IND Application for PBML04 for Treatment of Metachromatic Leukodystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","year":"2022","type":"Not Applicable","leadProduct":"PBML04","moa":"ARSA gene","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Passage Bio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Passage Bio \/ University of Pennsylvania","highestDevelopmentStatusID":"5","companyTruncated":"Passage Bi.."},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AVROBIO Receives Orphan Drug Designation from the U.S. Food and Drug Administration for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","year":"2022","type":"Not Applicable","leadProduct":"AVR-RD-05","moa":"IDS","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Avrobio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Avrobio \/ .."},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Cortexyme Announces Agreement to Acquire Novosteo","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Acquisition","leadProduct":"NOV004","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Quince Therapeutics \/ Cortexyme","highestDevelopmentStatusID":"5","companyTruncated":"Quince The.."},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Cortexyme Successfully Completes Acquisition of Novosteo","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Acquisition","leadProduct":"NOV004","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Quince Therapeutics \/ Cortexyme","highestDevelopmentStatusID":"5","companyTruncated":"Quince The.."},{"orgOrder":0,"company":"Cure Rare","sponsor":"Charles River Laboratories, Inc","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","year":"2022","type":"Not Applicable","leadProduct":"CRD-TMH-001","moa":"Dystrophin protein","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Cure Rare","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Cure Rare \/ Charles River Laboratories","highestDevelopmentStatusID":"5","companyTruncated":"Cure Rare .."},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quince Therapeutics to Present at Upcoming Scientific Meetings","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2022","year":"2022","type":"Not Applicable","leadProduct":"NOV004","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Quince Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Quince The.."},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quince Therapeutics Presents Preclinical Data at OI 2022 Demonstrating Potential of a Targeted Anabolic for Accelerated Bone Healing of Osteogenesis Imperfecta Fractures","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2022","year":"2022","type":"Not Applicable","leadProduct":"NOV004","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Quince Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Quince The.."},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Immusoft Announces FDA Clearance of IND Application for ISP-001 for MPS I, the First Engineered B Cell Therapy to Enter into Clinical Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","year":"2022","type":"Not Applicable","leadProduct":"Iduronicrin Genleukocel-T","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Immusoft Corporation","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Immusoft Corporation \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Immusoft C.."},{"orgOrder":0,"company":"Praxis Precision Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Praxis Precision Medicines Provides Updates on Clinical Stage Pediatric Epilepsy Programs","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","year":"2022","type":"Not Applicable","leadProduct":"PRAX-222","moa":"SCN2A gene","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Praxis Precision Medicines","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Praxis Precision Medicines \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Praxis Pre.."},{"orgOrder":0,"company":"Quince Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quince Therapeutics Presents Preclinical Data at ASBMR 2022 Demonstrating Application of Bone-targeting Platform for Spinal Fusion and Bone Cancer Indications","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"September 2022","year":"2022","type":"Not Applicable","leadProduct":"NOV004","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Quince Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Quince Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Quince The.."},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AVROBIO Announces Neuronopathic Mucopolysaccharidosis Type II (nMPS-II) or Hunter Syndrome Clinical Trial Application (CTA) Accepted by U.K. Regulatory Agency to Initiate Pediatric Phase 1\/2 Gene Therapy Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","year":"2022","type":"Not Applicable","leadProduct":"AVR-RD-05","moa":"IDS","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Avrobio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Avrobio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Avrobio \/ .."},{"orgOrder":0,"company":"Praxis Bioresearch","sponsor":"Invitae","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Invitae\u2019s Real-World Ciitizen Data Utilized in Praxis Precision Medicines\u2019 PRAX-222 IND Filing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","year":"2022","type":"Not Applicable","leadProduct":"PRAX-222","moa":"SCN2A gene","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Praxis Bioresearch","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Praxis Bioresearch \/ Invitae","highestDevelopmentStatusID":"5","companyTruncated":"Praxis Bio.."},{"orgOrder":0,"company":"Ray Therapeutics","sponsor":"Forge Biologics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Ray Therapeutics and Forge Biologics Expand Their Viral Vector cGMP Partnership to Encompass Plasmid DNA Manufacturing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2022","year":"2022","type":"Collaboration","leadProduct":"RTx-015","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Ray Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Ray Therapeutics \/ Forge Biologics","highestDevelopmentStatusID":"5","companyTruncated":"Ray Therap.."},{"orgOrder":0,"company":"Vita Therapeutics","sponsor":"Cambrian BioPharma","pharmaFlowCategory":"D","amount":"$31.0 million","upfrontCash":"Undisclosed","newsHeadline":"Vita Therapeutics Closes $31 Million Series B Financing to Develop Cell Therapies for Neuromuscular Diseases and Cancers","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","year":"2022","type":"Series B Financing","leadProduct":"VTA-100","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vita Therapeutics","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Vita Therapeutics \/ Cambrian BioPharma","highestDevelopmentStatusID":"5","companyTruncated":"Vita Thera.."},{"orgOrder":0,"company":"Allomek","sponsor":"Camac Partners","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Pasithea Therapeutics Acquires AlloMek Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","year":"2022","type":"Acquisition","leadProduct":"CIP-137401","moa":"MEK1\/2 receptor","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Allomek","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Allomek \/ Camac Partners","highestDevelopmentStatusID":"5","companyTruncated":"Allomek \/ .."},{"orgOrder":0,"company":"TeraImmune","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"TeraImmune Receives FDA IND Clearance to Initiate Phase 1\/2a Clinical Trial of TI-168 for Treatment of Hemophilia A with Refractory Inhibitors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","year":"2022","type":"Not Applicable","leadProduct":"TI-168","moa":"Factor VIII","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"TeraImmune","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"TeraImmune \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"TeraImmune.."},{"orgOrder":0,"company":"Jaguar Health","sponsor":"Napo Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Medicines Agency Grants Orphan Drug Designation for Crofelemer for Microvillus Inclusion Disease (MVID), a Second Rare Disease Indication","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"","productType":"Small molecule","productStatus":"Approved","date":"October 2022","year":"2022","type":"Not Applicable","leadProduct":"Crofelemer","moa":"CFTR","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Jaguar Health","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Tablet, Delayed Release","sponsorNew":"Jaguar Health \/ Napo Therapeutics","highestDevelopmentStatusID":"5","companyTruncated":"Jaguar Hea.."},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"EIC Accelerator","pharmaFlowCategory":"D","amount":"$13.7 million","upfrontCash":"Undisclosed","newsHeadline":"Arthex Biotech Selected for EIC Accelerator Award of up to 14 Million Euros","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2022","year":"2022","type":"Funding","leadProduct":"ATX-01","moa":"MBNL protein","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ARTHEx Biotech","amount2":0.01,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.01,"dosageForm":"","sponsorNew":"ARTHEx Biotech \/ EIC Accelerator","highestDevelopmentStatusID":"5","companyTruncated":"ARTHEx Bio.."},{"orgOrder":0,"company":"Juvena Therapeutics","sponsor":"Mubadala Capital","pharmaFlowCategory":"D","amount":"$41.0 million","upfrontCash":"Undisclosed","newsHeadline":"Juvena Therapeutics Secures $41 Million to Accelerate the Discovery and Development of Biologics for Chronic and Age-Related Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","year":"2022","type":"Series A Financing","leadProduct":"JUV-161","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Juvena Therapeutics","amount2":0.040000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"Juvena Therapeutics \/ Mubadala Capital","highestDevelopmentStatusID":"5","companyTruncated":"Juvena The.."},{"orgOrder":0,"company":"Wellbeing Digital Sciences","sponsor":"Nova Mentis Life Science","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Wellbeing Subsidiary KGK Science Enters into Research Contract With Nova Mentis for Phase II A Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"Approved","date":"November 2022","year":"2022","type":"Agreement","leadProduct":"Psilocybine","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Wellbeing Digital Sciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Wellbeing Digital Sciences \/ Nova Mentis Life Science","highestDevelopmentStatusID":"5","companyTruncated":"Wellbeing .."},{"orgOrder":0,"company":"DepYmed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"DepYmed Receives European Medicines Agency Orphan Drug Designation for Its Lead Clinical Candidate for the Treatment of Rett Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"DepYmed","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"DepYmed \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"DepYmed \/ .."},{"orgOrder":0,"company":"Wellbeing Digital Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Wellbeing Subsidiary KGK Science Submits CTA for Phase IIA Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"Approved","date":"November 2022","year":"2022","type":"Not Applicable","leadProduct":"Psilocybine","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Wellbeing Digital Sciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Wellbeing Digital Sciences \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Wellbeing .."},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Not Applicable","leadProduct":"VX-522","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Vertex Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Powder for Inhalation","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Vertex Pha.."},{"orgOrder":0,"company":"ARTHEx Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arthex Announces Regulatory Milestones Met in Its Program to Develop ATX-01 in Myotonic Dystrophy Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Not Applicable","leadProduct":"ATX-01","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"ARTHEx Biotech","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"ARTHEx Biotech \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"ARTHEx Bio.."},{"orgOrder":0,"company":"NMD PHARMA","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NMD Pharma Receives IND Clearance to Start a Phase II Clinical Trial of NMD670 for the Treatment of Symptoms of Spinal Muscular Atrophy Type 3","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Not Applicable","leadProduct":"NMD670","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"NMD PHARMA","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"NMD PHARMA \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"NMD PHARMA.."},{"orgOrder":0,"company":"Neurophth Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurophth Receives IND Clearance from FDA for AAV-ND1 Gene Therapy of LHON","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Not Applicable","leadProduct":"rAAV2-ND1","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Neurophth Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Neurophth Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Neurophth .."},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"REGENXBIO Reports Update on Advancement of Programs for CLN2 Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Not Applicable","leadProduct":"RGX-181","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Regenxbio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Regenxbio .."},{"orgOrder":0,"company":"TeraImmune","sponsor":"National Institute of Allergy and Infectious Diseases","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"TeraImmune Extends CRADA for Development of Treg Expansion for Clinical Use","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","year":"2023","type":"Agreement","leadProduct":"TI-168","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"TeraImmune","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"TeraImmune \/ National Institute of Allergy and Infectious Diseases","highestDevelopmentStatusID":"5","companyTruncated":"TeraImmune.."},{"orgOrder":0,"company":"Pasithea Therapeutics","sponsor":"WuXi AppTec","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Pasithea Therapeutics Signs CMC Development and Manufacturing Agreement for the Production of PAS-004","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2023","year":"2023","type":"Agreement","leadProduct":"CIP-137401","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Pasithea Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Pasithea Therapeutics \/ WuXi AppTec Inc.","highestDevelopmentStatusID":"5","companyTruncated":"Pasithea T.."},{"orgOrder":0,"company":"Disc medicine","sponsor":"Mabwell","pharmaFlowCategory":"D","amount":"$422.5 million","upfrontCash":"$10.0 million","newsHeadline":"Disc Medicine Announces Exclusive Licensing Agreement with Mabwell Therapeutics for Novel Anti-TMPRSS6 Monoclonal Antibodies to Modulate Iron Homeostasis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","year":"2023","type":"Licensing Agreement","leadProduct":"MWTX-003","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Disc medicine","amount2":0.41999999999999998,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.41999999999999998,"dosageForm":"Injectable\/Injection","sponsorNew":"Disc medicine \/ Mabwell","highestDevelopmentStatusID":"5","companyTruncated":"Disc medic.."},{"orgOrder":0,"company":"AceLink Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AceLink Presents Preclinical Data on AL00804 at WORLD Symposium","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","year":"2023","type":"Not Applicable","leadProduct":"AL00804","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"AceLink Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"AceLink Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"AceLink Th.."},{"orgOrder":0,"company":"Jaguar Health","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to Jaguar Health for Crofelemer for Microvillus Inclusion Disease (MVID), a Second Rare Disease Indication in the US","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Small molecule","productStatus":"Approved","date":"February 2023","year":"2023","type":"Not A
Market Place