[{"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","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Prevail Therapeutics"},{"company":"Aeglea BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeglea BioTherapeutics Submits CTA Application for its Novel Engineered Human Enzyme Designed to Treat Homocystinuria (ACN00177)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Aeglea BioTherapeutics"},{"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","url1":"","url2":"","graph1":"Large molecule","graph2":"GeneTx Biotherapeutics"},{"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","url1":"","url2":"","graph1":"Small molecule","graph2":"Emerald Health Pharmaceuticals"},{"company":"Passage Bio","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$125.0 million","upfrontCash":"Undisclosed","newsHeadline":"Gene Therapy Developer Passage Bio Files for $125M IPO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Passage Bio"},{"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","url1":"","url2":"","graph1":"Undisclosed","graph2":"Sigilon Therapeutics"},{"company":"Arrowhead Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1\/2a Study of ARO-ENaC for Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Arrowhead Pharmaceuticals"},{"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Completion of IND-enabling Safety Studies for FTX-6058 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Fulcrum Therapeutics"},{"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":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"DiNAQOR AG"},{"company":"LogicBio Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LogicBio Therapeutics to Present New Data on GeneRide\u2122 Platform and Next Generation Capsid Development Program at ASGCT 2020\r\n","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"LogicBio Therapeutics"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Inozyme Pharma"},{"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","url1":"","url2":"","graph1":"Large molecule","graph2":"AmideBio"},{"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","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"LogicBio Therapeutics"},{"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BioMarin Pharmaceutical"},{"company":"LogicBio Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LogicBio Therapeutics Provides Update on FDA Review of LB-001 NDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"LogicBio Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Taysha Gene Therapies"},{"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Passage Bio Orphan Drug Designation for PBGM01 for Treatment of Infantile GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Passage Bio"},{"company":"4D Molecular Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"4D Molecular Therapeutics Announces Upcoming Oral and Poster Presentations at the American Society of Gene and Cell Therapy Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"4D Molecular Therapeutics"},{"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Passage Bio Receives Rare Pediatric Disease Designation for PBGM01 for Patients with GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Passage Bio"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Beam Therapeutics"},{"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Presents Pre-Clinical Data from a Study of EDIT-301 with Sickle Patient Cells for Potential Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Editas Medicine"},{"company":"4D Molecular Therapeutics","sponsor":"Viking Global Investors","pharmaFlowCategory":"D","amount":"$75.0 million","upfrontCash":"Undisclosed","newsHeadline":"4D Molecular Therapeutics Raises $75 Million in Series C Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"4D Molecular Therapeutics"},{"company":"HemoShear Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"HemoShear Therapeutics Receives FDA Clearance of IND for Phase 2 Study of its Investigational Drug HST5040","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"HemoShear Therapeutics"},{"company":"Inozyme Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$86.0 million","upfrontCash":"Undisclosed","newsHeadline":"Rare Disease Biotech Inozyme Pharma Files for an $86 million IPO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Inozyme Pharma"},{"company":"Azzur Group","sponsor":"Editas Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Editas Medicine and Azzur Group Announce a Multi-year Agreement for EDIT-301 and EDIT-201 Manufacturing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Azzur Group"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurogene"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alexion Pharmaceuticals"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurogene"},{"company":"Forge Biologics","sponsor":"Perceptive Xontogeny Venture Fund","pharmaFlowCategory":"D","amount":"$40.0 million","upfrontCash":"Undisclosed","newsHeadline":"Forge Debuts with $40M and Tim Miller at the Helm","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Forge Biologics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Abeona Therapeutics"},{"company":"4D Molecular Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"4D Molecular Therapeutics Receives FDA Fast Track Designation for 4D-310 Gene Therapy for Treatment of Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"4D Molecular Therapeutics"},{"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":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Peptide","graph2":"AmideBio"},{"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Receives Rare Pediatric Disease Designation For EDIT-301 For The Treatment Of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Editas Medicine"},{"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Taysha Gene Therapies Receives Orphan Drug Designation and Rare Pediatric Disease Designation for TSHA-101 for GM2 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Taysha Gene Therapies"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Abeona Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Graphite Bio"},{"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Preclinical Proof-of-Concept Data for FTX-6058 at the Virtual Annual Sickle Cell Disease Research & Educational Symposium and 43rd National Sickle Cell Disease Scientific Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Fulcrum Therapeutics"},{"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Presents New Preclinical Data Showing Persistent In Vivo Editing and Durability of Effect Following CRISPR\/Cas9-Based Treatment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Fulcrum Therapeutics"},{"company":"Voyager Therapeutics, Inc","sponsor":"Sanofi","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Voyager Therapeutics Provides Regulatory Update On VY-HTT01 Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Voyager Therapeutics, Inc"},{"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Passage Bio Announces Publication of Preclinical Data Supporting Advancement of PBGM01 into Clinic","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Passage Bio"},{"company":"SparingVision","sponsor":"4BIO Capital","pharmaFlowCategory":"D","amount":"$51.9 million","upfrontCash":"Undisclosed","newsHeadline":"SparingVision Raises \u20ac44.5 Million and Appoints St\u00e9phane Boissel\n as Chief Executive Officer","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"},{"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","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"University of North Carolina"},{"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":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Allomek"},{"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Passage Bio Receives MHRA Clinical Trial Authorization for PBGM01 for Treatment of GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Passage Bio"},{"company":"Graphite Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Graphite Bio Receives IND Clearance to Initiate Clinical Trial for Next-Generation Gene Editing Therapy GPH101 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Graphite Bio"},{"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":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"WuXi Biologics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurogene"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Sigilon Therapeutics"},{"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Receives MHRA and Research Ethics Committee Approvals to Initiate the Gene Therapy Clinical Trial in the UK with LYS-GM101","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"},{"company":"EdiGene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"China NMPA Approves EdiGene\u2019s IND Application of CRISPR\/Cas 9 Gene-Editing Therapy, ET-01 to Treat Transfusion Dependent \u00df-Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"EdiGene"},{"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":"","productType":"Peptide","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Peptide","graph2":"French National Institute of Health and Medical Research"},{"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":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Chinook Therapeutics"},{"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":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Rescindo Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Ultragenyx Pharmaceutical"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"DTx Pharma"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Adverum Biotechnologies"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"PYC Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Agtc"},{"company":"Hillhurst Biopharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Hillhurst Biopharmaceuticals Receives Study May Proceed Letter From US FDA to Initiate Phase 1 Clinical Trial of HBI-002","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Hillhurst Biopharmaceuticals"},{"company":"Inozyme Pharma","sponsor":"Thomas Jefferson University","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme Pharma and Thomas Jefferson University to Present Preclinical Data Suggesting Utility of INZ-701 for ABCC6 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Inozyme Pharma"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Inozyme Pharma"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Enzyvant"},{"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":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"SpliSense"},{"company":"Verve Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$266.7 million","upfrontCash":"Undisclosed","newsHeadline":"Gene Editing Biotech Verve Therapeutics Files for a $100 Million IPO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Verve Therapeutics"},{"company":"Synlogic","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Synlogic Presents Additional Preclinical Data on Therapeutic Candidates SYNB1934 for Phenylketonuria and SYNB8802 for Enteric Hyperoxaluria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Synlogic"},{"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":"INDIA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Vital Therapeutics & Formulations Pvt Ltd"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Sigilon Therapeutics"},{"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":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"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":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Polaryx Therapeutics"},{"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":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"September 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"ADARx Pharmaceuticals"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Precision BioSciences"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"NeuExcell therapeutics"},{"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics Receives Authorization to Initiate Phase 1\/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"company":"Graphite Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Graphite Bio Presents Preclinical Data Supporting GPH101 in Sickle Cell Disease at 49th Annual Sickle Cell Disease Association of America National Convention","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Graphite Bio"},{"company":"Tenaya Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Tenaya Therapeutics to Present Preclinical Data on Its Gene Therapy Programs at the ESGCT 28th Annual Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"October 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Tenaya Therapeutics"},{"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Pharmaceuticals CAN103 Investigational New Drug Application Approved in China for Treatment of Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"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":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Healx"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"ContraFect Corporation"},{"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":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"University of L'Aquila"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"GeneVentiv Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Avrobio"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"ContraFect Corporation"},{"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":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Immusoft Corporation"},{"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":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Amarna Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Beam Therapeutics"},{"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":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Neurophth Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Beam Therapeutics"},{"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":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Ractigen Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Sigilon Therapeutics"},{"company":"Dyne Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dyne Therapeutics Announces FDA Clinical Hold on IND Application for DYNE-251 in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Dyne Therapeutics"},{"company":"Jnana Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Jnana Therapeutics To Advance JNT-517 As Development Candidate And Potential First-In-Class Oral Treatment For PKU","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Jnana Therapeutics"},{"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stealth BioTherapeutics Showcases New Duchenne Muscular Dystrophy Nonclinical Data at the 2022 Muscular Dystrophy Association and Clinical Scientific Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Stealth Biotherapeutics"},{"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":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Peptide","graph2":"PepGen"},{"company":"Sionna Therapeutics","sponsor":"OrbiMed","pharmaFlowCategory":"D","amount":"$111.0 million","upfrontCash":"Undisclosed","newsHeadline":"Sionna Therapeutics Launches with $111 Million Series B Financing to Advance Pipeline of Novel Small Molecules with the Potential to Fully Restore CFTR Function in Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Sionna Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Editas Medicine"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Precision BioSciences"},{"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Announces Presentations at ARVO 2022 Annual Meeting and ASGCT 25th Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atsena Therapeutics"},{"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":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"DepYmed"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Opus Genetics"},{"company":"Aro Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aro Biotherapeutics to Present New Preclinical Data Highlighting the Potential of Centyrin-siRNA Conjugates for the Treatment of Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Aro Biotherapeutics"},{"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Announces FDA Acceptance of Investigational New Drug (IND) Application for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"company":"ASC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ASC Therapeutics Announces Podium and Poster Presentations of IND-Enabling Studies for ASC618, a Second-Generation Gene Therapy for Hemophilia A, at the 25th ASGCT Meeting, May 16-19, 2022 in Washington, D.C.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"ASC Therapeutics"},{"company":"Mustang Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mustang Bio Announces Updated Interim Data on X-Linked Severe Combined Immunodeficiency Treatment with Lentiviral Vector Gene Therapy Support Upcoming Multicenter Pivotal Phase 2 Trial for MB-107","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Mustang Bio"},{"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":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Neuronascent"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Passage Bio"},{"company":"Dyne Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dyne Therapeutics Announces Clearance of Clinical Trial Application for DYNE-101 for the Treatment of Myotonic Dystrophy Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Dyne Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Avrobio"},{"company":"Astria Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astria Therapeutics Announces FDA Clearance of IND Application for STAR-0215, a Monoclonal Antibody Inhibitor of Plasma Kallikrein for Treatment of Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Astria Therapeutics"},{"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":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Quince Therapeutics"},{"company":"Aro Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aro Biotherapeutics Receives FDA Orphan Drug Designation for ABX1100 for the Treatment of Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Aro Biotherapeutics"},{"company":"Dyne Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dyne Therapeutics Announces \u201cBreakthrough Article\u201d Publication of Duchenne Muscular Dystrophy Program Data in Nucleic Acids Research","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Dyne Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Cure Rare"},{"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":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Quince Therapeutics"},{"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":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Quince Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Immusoft Corporation"},{"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":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Praxis Precision Medicines"},{"company":"SparingVision","sponsor":"Jeito Capital","pharmaFlowCategory":"D","amount":"$75.0 million","upfrontCash":"Undisclosed","newsHeadline":"SparingVision Raises \u20ac75 Million Series B to Continue Building World-Leading Portfolio of Genomic Medicines for Ocular Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"},{"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":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Quince Therapeutics"},{"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":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Avrobio"},{"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":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Praxis Bioresearch"},{"company":"Aro Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aro Biotherapeutics Receives FDA Rare Pediatric Drug Designation for ABX1100 for the Treatment of Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Aro Biotherapeutics"}]
Details:
PRAX-222 is an ASO designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations.
Lead Product(s):
PRAX-222
Therapeutic Area: Genetic Disease
Product Name: PRAX-222
Highest Development Status: IND Enabling
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Invitae
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 20, 2022
Details:
ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in muscle, for the treatment of Pompe disease. ABX1100 is a novel approach that aims to reduce glycogen buildup in the muscles.
Lead Product(s):
ABX1100
Therapeutic Area: Genetic Disease
Product Name: ABX1100
Highest Development Status: IND Enabling
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 15, 2022
Details:
Proceeds from the financing will be used to fund the first-in-human trials of the Company’s two lead gene-independent assets, SPVN06 and SPVN20, as well as the development of genome editing assets through its collaboration with Intellia Therapeutics.
Lead Product(s):
SPVN06
Therapeutic Area: Genetic Disease
Product Name: SPVN06
Highest Development Status: IND Enabling
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Jeito Capital
Deal Size: $75.0 million
Upfront Cash: Undisclosed
Deal Type: Series B Financing
September 14, 2022
Details:
The gene therapy, which AVROBIO refers to as AVR-RD-05, is designed to transduce autologous (a patient’s own) HSCs ex vivo with a lentiviral vector encoding a brain-targeted iduronate-2-sulfatase (IDS) enzyme, which is deficient in these patients.
Lead Product(s):
AVR-RD-05
Therapeutic Area: Genetic Disease
Product Name: AVR-RD-05
Highest Development Status: IND Enabling
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 14, 2022
Details:
In preclinical studies, NOV004 concentrated at spinal fusion sites to demonstrate accelerated repair in treated animals which resulted in improved blinded radiographic fusion scores compared to animals treated with current standard of care, bone morphogenic protein-2.
Lead Product(s):
NOV004
Therapeutic Area: Genetic Disease
Product Name: NOV004
Highest Development Status: IND Enabling
Product Type: Peptide
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 12, 2022
Details:
PRAX-222 is an ASO designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-onset SCN2A DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations.
Lead Product(s):
PRAX-222
Therapeutic Area: Genetic Disease
Product Name: PRAX-222
Highest Development Status: IND Enabling
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 07, 2022
Details:
ISP-001 (iduronicrin genleukocel-T) represents the first product candidate developed using the company’s proprietary technique for the treatment of mucopolysaccharidosis type I (MPS I).
Lead Product(s):
Iduronicrin Genleukocel-T
Therapeutic Area: Genetic Disease
Product Name: ISP-001
Highest Development Status: IND Enabling
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 01, 2022
Details:
NOV004 was prepared by synthesizing an abaloparatide-like peptide conjugated to a hydroxyapatite-homing acidic oligopeptide to create a high affinity to bone fractures.
Lead Product(s):
NOV004
Therapeutic Area: Genetic Disease
Product Name: NOV004
Highest Development Status: IND Enabling
Product Type: Peptide
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 30, 2022
Details:
The company’s lead compound NOV004 is an anabolic peptide engineered to precisely target and concentrate at the bone fracture site, which preclinical studies demonstrate result in rapid increases in bone density, strength, and healing directly at the site of bone fracture
Lead Product(s):
NOV004
Therapeutic Area: Genetic Disease
Product Name: NOV004
Highest Development Status: IND Enabling
Product Type: Peptide
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 25, 2022
Details:
The drug, named CRD-TMH-001, treats muscle promoter and exon 1 mutations on the dystrophin gene, to stop the progression of Duchenne muscular dystrophy in a single-patient dosing.
Lead Product(s):
CRD-TMH-001
Therapeutic Area: Genetic Disease
Product Name: CRD-TMH-001
Highest Development Status: IND Enabling
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Charles River Laboratories, Inc
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
August 11, 2022
Market Place
News Type
Upload your Marketing & Sales content on your company Virtual Booth, click HERE
