[{"orgOrder":0,"company":"Light Chain Bioscience","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Novimmune unit nabs Takeda milestone payment for hemophilia work","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"SWITZERLAND","productType":"Large molecule","productStatus":"Undisclosed","date":"February 2020","year":"2020","type":"Collaboration","leadProduct":"Bispecific antibodies","moa":"Factor VIII","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Light Chain Bioscience","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Light Chain Bioscience \/ Takeda","highestDevelopmentStatusID":"2","companyTruncated":"Light Chain Bioscience \/ Takeda"},{"orgOrder":0,"company":"Nimbus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nimbus Therapeutics Announces Expansion of its Drug Discovery Pipeline Across Oncology, Immunology and Metabolism","therapeuticArea":"Endocrinology","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"June 2020","year":"2020","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Nimbus Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Nimbus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Nimbus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Gero","sponsor":"Bulba Ventures","pharmaFlowCategory":"D","amount":"$2.2 million","upfrontCash":"Undisclosed","newsHeadline":"Gero, a Singapore-based Biotech Startup raises $2.2M in Series A Funding","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"SINGAPORE","productType":"Undisclosed","productStatus":"Undisclosed","date":"August 2020","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Gero","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Gero \/ Bulba Ventures","highestDevelopmentStatusID":"2","companyTruncated":"Gero \/ Bulba Ventures"},{"orgOrder":0,"company":"Evotec","sponsor":"Centogene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Evotec and Centogene Expand Collaboration into Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"August 2020","year":"2020","type":"Expanded Collaboration","leadProduct":"Induced pluripotent stem cell","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Evotec","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Evotec \/ CENTOGENE","highestDevelopmentStatusID":"2","companyTruncated":"Evotec \/ CENTOGENE"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","amount":"$455.0 million","upfrontCash":"$75.0 million","newsHeadline":"Moderna and Vertex Establish New Collaboration to Treat Cystic Fibrosis Using Gene Editing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"September 2020","year":"2020","type":"Collaboration","leadProduct":"LNP based mRNA Therapeutics","moa":"CFTR","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Moderna Therapeutics","amount2":0.46000000000000002,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.46000000000000002,"dosageForm":"","sponsorNew":"Moderna Therapeutics \/ Vertex","highestDevelopmentStatusID":"2","companyTruncated":"Moderna Therapeutics \/ Vertex"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"MyoKardia","pharmaFlowCategory":"D","amount":"$465.0 million","upfrontCash":"$12.5 million","newsHeadline":"MyoKardia and Fulcrum Therapeutics Announce Multi-Target Collaboration to Discover Novel Targeted Therapies for Genetic Cardiomyopathies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"July 2020","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Fulcrum Therapeutics","amount2":0.46999999999999997,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.46999999999999997,"dosageForm":"","sponsorNew":"Fulcrum Therapeutics \/ MyoKardia","highestDevelopmentStatusID":"2","companyTruncated":"Fulcrum Therapeutics \/ MyoKardia"},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"Foundation to Fight H-ABC","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Foundation to Fight H-ABC, University of Massachusetts Medical School and Yale University Initiate Gene Therapy Study Targeting Cure for Rare Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","year":"2020","type":"Agreement","leadProduct":"AAV-based gene","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"University of Massachusetts Medical School","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"University of Massachusetts Medical School \/ Foundation to Fight H-ABC","highestDevelopmentStatusID":"2","companyTruncated":"University of Massachusetts Medical School \/ Foundation to Fight H-ABC"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Taysha Gene Therapies Gets Rare Pediatric Disease and Orphan Drug Designations for TSHA-105 to Treat Epilepsy Caused by SLC13A5 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","year":"2021","type":"Not Applicable","leadProduct":"AAV9-based gene therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Taysha Gene Therapies","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Taysha Gene Therapies \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Taysha Gene Therapies \/ Not Applicable"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"Taysha Gene Therapies","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Taysha Gene Therapies and UT Southwestern Medical Center Launch Innovation Fund to Accelerate Advancement of AAV Gene Therapies for CNS","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","year":"2021","type":"Expanded Collaboration","leadProduct":"TSHA-112","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"UT Southwestern Medical Center","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"UT Southwestern Medical Center \/ Taysha Gene Therapies","highestDevelopmentStatusID":"2","companyTruncated":"UT Southwestern Medical Center \/ Taysha Gene Therapies"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Bill & Melinda Gates Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Novartis and the Gates Foundation Collaborate to Discover and Develop an Accessible in Vivo Gene Therapy for Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2021","year":"2021","type":"Collaboration","leadProduct":"Gene Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Bill & Melinda Gates Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Bill & Melinda Gates Foundation"},{"orgOrder":0,"company":"Sanofi","sponsor":"Global Blood Therapeutics","pharmaFlowCategory":"D","amount":"$353.0 million","upfrontCash":"Undisclosed","newsHeadline":"GBT Expands Sickle Cell Disease Pipeline with Exclusive In-license of Two Novel Small Molecule Programs from Sanofi S.A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"March 2021","year":"2021","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sanofi","amount2":0.34999999999999998,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.34999999999999998,"dosageForm":"","sponsorNew":"Sanofi \/ Global Blood Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Sanofi \/ Global Blood Therapeutics"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Renaissance Capital","pharmaFlowCategory":"D","amount":"$228.0 million","upfrontCash":"Undisclosed","newsHeadline":"Preclinical Genetic Disease Biotech Design Therapeutics Sets Terms for $228 Million IPO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2021","year":"2021","type":"Public Offering","leadProduct":"Gene targeted chimeras","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Design Therapeutics","amount2":0.23000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.23000000000000001,"dosageForm":"","sponsorNew":"Design Therapeutics \/ Renaissance Capital","highestDevelopmentStatusID":"2","companyTruncated":"Design Therapeutics \/ Renaissance Capital"},{"orgOrder":0,"company":"PAQ Therapeutics","sponsor":"Sherpa Healthcare","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"PAQ Therapeutics Launches with $30 Million Series A to Develop Novel Therapies through Autophagy-Dependent Degradation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2021","year":"2021","type":"Series A Financing","leadProduct":"Autophagosome-tethering Compound Based Therapy","moa":"Ubiquitin-proteosome pathway","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"PAQ Therapeutics","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"PAQ Therapeutics \/ Sherpa Healthcare","highestDevelopmentStatusID":"2","companyTruncated":"PAQ Therapeutics \/ Sherpa Healthcare"},{"orgOrder":0,"company":"Shape Therapeutics","sponsor":"Decheng Capital","pharmaFlowCategory":"D","amount":"$112.0 million","upfrontCash":"Undisclosed","newsHeadline":"Shape Therapeutics Secures $112 Million in Series B Financing to Advance Breakthrough RNA Technology Platforms to Democratize Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","year":"2021","type":"Series B Financing","leadProduct":"RNA Therapeutics","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Shape Therapeutics","amount2":0.11,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.11,"dosageForm":"","sponsorNew":"Shape Therapeutics \/ Decheng Capital","highestDevelopmentStatusID":"2","companyTruncated":"Shape Therapeutics \/ Decheng Capital"},{"orgOrder":0,"company":"Exicure","sponsor":"Ipsen","pharmaFlowCategory":"D","amount":"$1,020.0 million","upfrontCash":"$20.0 million","newsHeadline":"ipsen and Exicure Enter Into Exclusive Collaboration Targeting Rare Neurodegenerative Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"August 2021","year":"2021","type":"Collaboration","leadProduct":"SNA-based Therapeutics","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Exicure","amount2":1.02,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":1.02,"dosageForm":"","sponsorNew":"Exicure \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Exicure \/ Ipsen"},{"orgOrder":0,"company":"Tottori University","sponsor":"GC Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"GC Pharma and Tottori University Enter Research Collaboration and License Agreement for Development of GM1 Gangliosidosis Chaperone Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2021","year":"2021","type":"Collaboration","leadProduct":"Chaperone Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Tottori University","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Tottori University \/ GC Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Tottori University \/ GC Pharma"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","amount":"$1,300.0 million","upfrontCash":"$50.0 million","newsHeadline":"ProQR Announces Axiomer\u00ae RNA Editing Licensing and Research Collaboration with Lilly","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"Undisclosed","date":"September 2021","year":"2021","type":"Collaboration","leadProduct":"Editing Oligonucleotides","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":1.3,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":1.3,"dosageForm":"","sponsorNew":"ProQR Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"McGill University","sponsor":"Clarus","pharmaFlowCategory":"D","amount":"$40.8 million","upfrontCash":"$0.4 million","newsHeadline":"Clarus Therapeutics and McGill University Announce Exclusive Worldwide Licensing Agreement To Develop and Commercialize Technology To Treat Rare Conditions Associated With Coenzyme-Q10 (Ubiquinone) Deficiencies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 2021","year":"2021","type":"Licensing Agreement","leadProduct":"Coenzyme Q10","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"McGill University","amount2":0.040000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.040000000000000001,"dosageForm":"Oral","sponsorNew":"McGill University \/ Clarus","highestDevelopmentStatusID":"2","companyTruncated":"McGill University \/ Clarus"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","amount":"$630.0 million","upfrontCash":"$30.0 million","newsHeadline":"Voyager Therapeutics Announces License Option Agreement with Pfizer for Next-Generation TRACER\u1d40\u1d39 AAV Capsids to Enable Neurologic and Cardiovascular Gene Therapy Programs","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","year":"2021","type":"Licensing Agreement","leadProduct":"AAV Capsids","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Voyager Therapeutics, Inc","amount2":0.63,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.63,"dosageForm":"","sponsorNew":"Voyager Therapeutics, Inc \/ Pfizer","highestDevelopmentStatusID":"2","companyTruncated":"Voyager Therapeutics, Inc \/ Pfizer"},{"orgOrder":0,"company":"Scriptr","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"CANbridge Pharmaceuticals Enters into Exclusive Worldwide Research Collaboration and Licensing Agreement with Scriptr Global for Stitchr\u2122 RNA Assembly Technology to Develop Gene Therapy Product Targeting Dystrophinopathies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"October 2021","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scriptr","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Scriptr \/ CANbridge Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"Scriptr \/ CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Flagship Pioneering","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","amount":"$110.0 million","upfrontCash":"Undisclosed","newsHeadline":"Pioneering Medicines, an Initiative of Flagship Pioneering, Establishes First-of-its-Kind Partnership with Cystic Fibrosis Foundation to Develop Breakthrough Treatments for CF","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"Undisclosed","date":"November 2021","year":"2021","type":"Partnership","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Flagship Pioneering","amount2":0.11,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.11,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Flagship Pioneering \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Flagship Pioneering \/ Cystic Fibrosis Foundation"},{"orgOrder":0,"company":"EdiGene","sponsor":"University of Wisconsin\u2013Madison","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"EdiGene Enters Research Collaboration with the University of Wisconsin-Madison on in Vivo RNA Editing Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","year":"2021","type":"Collaboration","leadProduct":"Short Engineered ADAR-recruiting RNAs","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"EdiGene","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"EdiGene \/ University of Wisconsin\u2013Madison","highestDevelopmentStatusID":"2","companyTruncated":"EdiGene \/ University of Wisconsin\u2013Madison"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"OMass Unveils Rich Drug Discovery Pipeline Targeting Intractable or Inadequately Drugged Membrane and Complex-bound Proteins","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"Undisclosed","date":"November 2021","year":"2021","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"LoQus23 Therapeutics","sponsor":"Dementia Discovery Fund","pharmaFlowCategory":"D","amount":"$9.4 million","upfrontCash":"Undisclosed","newsHeadline":"LoQus23 Therapeutics Exits Stealth with \u00a311.5 Million Seed Financing From the Dementia Discovery Fund and Novartis Venture Fund","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"November 2021","year":"2021","type":"Financing","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"LoQus23 Therapeutics","amount2":0.01,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.01,"dosageForm":"Oral","sponsorNew":"LoQus23 Therapeutics \/ Dementia Discovery Fund","highestDevelopmentStatusID":"2","companyTruncated":"LoQus23 Therapeutics \/ Dementia Discovery Fund"},{"orgOrder":0,"company":"Dyno Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","amount":"$1,618.0 million","upfrontCash":"$18.0 million","newsHeadline":"Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","year":"2021","type":"Collaboration","leadProduct":"AAV Capsids","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Dyno Therapeutics","amount2":1.6200000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":1.6200000000000001,"dosageForm":"Dosage Strength\/Form","sponsorNew":"Dyno Therapeutics \/ Astellas Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Dyno Therapeutics \/ Astellas Pharma"},{"orgOrder":0,"company":"Korro Bio","sponsor":"Eventide Asset Management","pharmaFlowCategory":"D","amount":"$116.0 million","upfrontCash":"Undisclosed","newsHeadline":"Korro Bio Completes $116M Series B Financing to Expand the Frontier of Genetic Medicine through its Pipeline of RNA Editing Programs","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"Undisclosed","date":"January 2022","year":"2022","type":"Series B Financing","leadProduct":"Genetic Medicine","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Korro Bio","amount2":0.12,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.12,"dosageForm":"","sponsorNew":"Korro Bio \/ Eventide Asset Management","highestDevelopmentStatusID":"2","companyTruncated":"Korro Bio \/ Eventide Asset Management"},{"orgOrder":0,"company":"Serotiny","sponsor":"Tessera Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Serotiny and Tessera Therapeutics Enter Into Collaboration to Engineer and Optimize Programmable Gene Writer Proteins","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2022","year":"2022","type":"Collaboration","leadProduct":"Genetic Medicine","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Serotiny","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Serotiny \/ Tessera Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Serotiny \/ Tessera Therapeutics"},{"orgOrder":0,"company":"Ceptur Therapeutics","sponsor":"venBio Partners","pharmaFlowCategory":"D","amount":"$75.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ceptur Therapeutics Launches with $75M Series A Financing to Advance RNA Therapeutics Based on Proprietary U1 Adaptor Technology","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","year":"2022","type":"Series A Financing","leadProduct":"Oligonucleotide-based RNA Therapeutics","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Ceptur Therapeutics","amount2":0.080000000000000002,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Ceptur Therapeutics \/ venBio Partners","highestDevelopmentStatusID":"2","companyTruncated":"Ceptur Therapeutics \/ venBio Partners"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Yarrow Biotechnology","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$115.0 million","newsHeadline":"ProQR Therapeutics and Yarrow Biotechnology, an RTW Investments, LP Incubated Company, Announce Exclusive Worldwide License and Discovery Collaboration for Undisclosed Target","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","year":"2021","type":"Collaboration","leadProduct":"Antisense Oligonucleotide Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"ProQR Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"ProQR Therapeutics \/ Yarrow Biotechnology","highestDevelopmentStatusID":"2","companyTruncated":"ProQR Therapeutics \/ Yarrow Biotechnology"},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"Barth Syndrome Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Scenic Biotech and the Barth Syndrome Foundation Announce Partnership to Explore Genetic Modifiers to a Find Tailored Treatment for the Complex Rare Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"March 2022","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scenic Biotech","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Scenic Biotech \/ Barth Syndrome Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Scenic Biotech \/ Barth Syndrome Foundation"},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"GV","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"OMass Therapeutics Raises $100 Million in Series B Financing to Progress Drug Pipeline in Immunology and Rare Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"April 2022","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0.10000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.10000000000000001,"dosageForm":"Inhalation","sponsorNew":"OMass Therapeutics \/ GV","highestDevelopmentStatusID":"2","companyTruncated":"OMass Therapeutics \/ GV"},{"orgOrder":0,"company":"Codexis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Codexis to Present Data Highlighting Three Gene Therapy Programs at the ASGCT 25th Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Codexis","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Codexis \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Codexis \/ Not Applicable"},{"orgOrder":0,"company":"Bit Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bit.bio Launches Huntington\u2019s Disease Human Cell Model","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Bit Bio \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Not Applicable"},{"orgOrder":0,"company":"LUCA Science","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"LUCA Science and Kyowa Kirin Announce Joint Research Agreement on Mitochondrial Disease Treatment With Novel Mitochondria Modality","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","year":"2022","type":"Collaboration","leadProduct":"Mitochondrial-based Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"LUCA Science","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"LUCA Science \/ Kyowa Kirin Co.","highestDevelopmentStatusID":"2","companyTruncated":"LUCA Science \/ Kyowa Kirin Co."},{"orgOrder":0,"company":"Bit Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"bit.bio Presents Data at ISSCR Demonstrating Their Platform Capability to Create Any Human Cell","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","year":"2022","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Bit Bio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Bit Bio \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Bit Bio \/ Not Applicable"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Baylor College of Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BridgeBio Pharma Announces Academic Collaboration with Baylor College of Medicine to Advance Potential Therapies to Treat Genetic Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"Undisclosed","date":"July 2022","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"BridgeBio Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"BridgeBio Pharma \/ Baylor College of Medicine","highestDevelopmentStatusID":"2","companyTruncated":"BridgeBio Pharma \/ Baylor College of Medicine"},{"orgOrder":0,"company":"Nimbus Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","amount":"$496.0 million","upfrontCash":"Undisclosed","newsHeadline":"Nimbus Therapeutics Announces Research Collaboration and License Agreement with Lilly for Small Molecule Activators of AMPK","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"October 2022","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"AMPK","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Nimbus Therapeutics","amount2":0.5,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.5,"dosageForm":"","sponsorNew":"Nimbus Therapeutics \/ Eli Lilly","highestDevelopmentStatusID":"2","companyTruncated":"Nimbus Therapeutics \/ Eli Lilly"},{"orgOrder":0,"company":"NeuBase Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"NeuBase Therapeutics Announces Gene Editing Research Agreement with Global Healthcare Company","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Peptide","productStatus":"Undisclosed","date":"October 2022","year":"2022","type":"Agreement","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"NeuBase Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"NeuBase Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"NeuBase Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Proteros","sponsor":"Adrestia Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Proteros and Adrestia Initiate Multi-Target Partnership to Discover First-In-Class Drugs for Intractable Genetic Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"Undisclosed","date":"October 2022","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Proteros","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Proteros \/ Adrestia Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Proteros \/ Adrestia Therapeutics"},{"orgOrder":0,"company":"Replay Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Replay Launches Big DNA HSV Gene Therapy Company, Telaria, Focused on Rare Skin Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","year":"2022","type":"Not Applicable","leadProduct":"SynHSV Gene Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Replay Bio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Replay Bio \/ Not Applicable","highestDevelopmentStatusID":"2","companyTruncated":"Replay Bio \/ Not Applicable"},{"orgOrder":0,"company":"Metrion Biosciences","sponsor":"KCNC1 Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Metrion Biosciences and The KCNC1 Foundation Collaborate on Development of New Ion Channel Modulators for Ultra-Rare Genetic Disorder","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Metrion Biosciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Metrion Biosciences \/ KCNC1 Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Metrion Biosciences \/ KCNC1 Foundation"},{"orgOrder":0,"company":"Exicure","sponsor":"Ipsen","pharmaFlowCategory":"D","amount":"$1,020.0 million","upfrontCash":"$20.0 million","newsHeadline":"Exicure Announces Termination of AbbVie and Ipsen Collaboration Agreements","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Termination","leadProduct":"SNA-based Therapeutic","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Exicure","amount2":1.02,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":1.02,"dosageForm":"","sponsorNew":"Exicure \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Exicure \/ Ipsen"},{"orgOrder":0,"company":"Sanegene Bio","sponsor":"Orbit Discovery","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orbit Discovery and SanegeneBio Collaborate to Identify Targeting Peptides for RNAi Drugs","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","year":"2023","type":"Collaboration","leadProduct":"RNAi-based Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sanegene Bio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Sanegene Bio \/ Orbit Discovery","highestDevelopmentStatusID":"2","companyTruncated":"Sanegene Bio \/ Orbit Discovery"},{"orgOrder":0,"company":"GERO.AI","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Longevity Biotech Gero Entered a Research Collaboration with Pfizer to Discover Potential Targets for Fibrotic Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"January 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"GERO.AI","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"GERO.AI \/ Pfizer Inc","highestDevelopmentStatusID":"2","companyTruncated":"GERO.AI \/ Pfizer Inc"},{"orgOrder":0,"company":"Gensaic","sponsor":"CF Foundation","pharmaFlowCategory":"D","amount":"$3.5 million","upfrontCash":"Undisclosed","newsHeadline":"Gensaic Receives Funding From the CF Foundation to Develop a Gene Therapy for People With Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","year":"2023","type":"Funding","leadProduct":"CFTR-based Gene Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Gensaic","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Gensaic \/ CF Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Gensaic \/ CF Foundation"},{"orgOrder":0,"company":"Myosana Therapeutics","sponsor":"John Ballantyne","pharmaFlowCategory":"D","amount":"$5.0 million","upfrontCash":"Undisclosed","newsHeadline":"Myosana Therapeutics Raises $5 Million in Seed Funding","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","year":"2023","type":"Financing","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Myosana Therapeutics","amount2":0.01,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.01,"dosageForm":"","sponsorNew":"Myosana Therapeutics \/ John Ballantyne","highestDevelopmentStatusID":"2","companyTruncated":"Myosana Therapeutics \/ John Ballantyne"},{"orgOrder":0,"company":"Spark Therapeutics, Inc","sponsor":"Neurochase","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Spark Therapeutics Enters Into Strategic Collaboration with Neurochase for Use of Proprietary Delivery Technology for CNS Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Spark Therapeutics, Inc","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Spark Therapeutics, Inc \/ Neurochase","highestDevelopmentStatusID":"2","companyTruncated":"Spark Therapeutics, Inc \/ Neurochase"},{"orgOrder":0,"company":"Nanite","sponsor":"CMT Research Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"CMT Research Foundation and Nanite Inc. Announce Partnership to Enhance Therapeutic Efficacy of Antisense Oligonucleotides in CMT1A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"Undisclosed","date":"February 2023","year":"2023","type":"Partnership","leadProduct":"Antisense Oligonucleotide","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Nanite","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Nanite \/ CMT Research Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Nanite \/ CMT Research Foundation"},{"orgOrder":0,"company":"Aitia","sponsor":"UCB Pharma S.A","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Aitia and UCB Announce Strategic Drug Discovery Collaboration in Huntington's Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"March 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Aitia","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Aitia \/ UCB Pharma S.A","highestDevelopmentStatusID":"2","companyTruncated":"Aitia \/ UCB Pharma S.A"},{"orgOrder":0,"company":"Kate Therapeutics","sponsor":"Westlake Village BioPartners","pharmaFlowCategory":"D","amount":"$51.0 million","upfrontCash":"Undisclosed","newsHeadline":"Kate Therapeutics Debuts with $51 Million Series A to Develop Next-generation Genetic Medicines to Treat Muscle and Heart Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2023","year":"2023","type":"Series A Financing","leadProduct":"AAV-based Gene Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Kate Therapeutics","amount2":0.050000000000000003,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.050000000000000003,"dosageForm":"","sponsorNew":"Kate Therapeutics \/ Westlake Village BioPartners","highestDevelopmentStatusID":"2","companyTruncated":"Kate Therapeutics \/ Westlake Village BioPartners"},{"orgOrder":0,"company":"IRBM","sponsor":"Merck & Co","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"IRBM Extends Peptide Therapeutics Collaboration with Merck & Co. Inc., Rahway NJ USA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"June 2023","year":"2023","type":"Expanded Collaboration","leadProduct":"Peptide Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"IRBM","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"IRBM \/ Merck & Co. Inc.","highestDevelopmentStatusID":"2","companyTruncated":"IRBM \/ Merck & Co. Inc."},{"orgOrder":0,"company":"Poolbeg Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Poolbeg Partners with a Nasdaq-Listed Biopharma","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Vaccine","productStatus":"New Molecular Entity","date":"October 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Poolbeg Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Poolbeg Pharma \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"Poolbeg Pharma \/ Undisclosed"},{"orgOrder":0,"company":"Genevant Sciences","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genevant Sciences to Collaborate with Novo Nordisk to Develop Gene Editing Treatment for Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Genevant Sciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Genevant Sciences \/ Novo Nordisk","highestDevelopmentStatusID":"2","companyTruncated":"Genevant Sciences \/ Novo Nordisk"},{"orgOrder":0,"company":"Fractyl Health","sponsor":"Michigan Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Fractyl Health Expands Landmark Academic-Industry Collaboration to Study Mechanisms Underlying GLP-1-Based Pancreatic Gene Therapy for Type 2 Diabetes and Obesity","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","year":"2023","type":"Collaboration","leadProduct":"Pancreatic Gene Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Fractyl Health","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Fractyl Health \/ Michigan Medicine","highestDevelopmentStatusID":"2","companyTruncated":"Fractyl Health \/ Michigan Medicine"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Genetic Cures for Kids","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Charles River and Genetic Cures for Kids Announce Gene Therapy Manufacturing Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Charles River Laboratories, Inc","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Charles River Laboratories, Inc \/ Genetic Cures for Kids","highestDevelopmentStatusID":"2","companyTruncated":"Charles River Laboratories, Inc \/ Genetic Cures for Kids"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","amount":"$1,240.0 million","upfrontCash":"$40.0 million","newsHeadline":"Scribe Therapeutics Expands In Vivo Collaboration with Sanofi to Second Target","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","year":"2024","type":"Expanded Collaboration","leadProduct":"CRISPR-based Therapy","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Scribe Therapeutics","amount2":1.24,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":1.24,"dosageForm":"","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"2","companyTruncated":"Scribe Therapeutics \/ Sanofi"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Filters
×
FILTER:
Company Name
Year
DEALS // DEV.
Country
Sponsor
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target