[{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Carmine Therapeutics","pharmaFlowCategory":"D","amount":"$900.0 million","upfrontCash":"Undisclosed","newsHeadline":"Takeda Partners up with Carmine Therapeutics to Develop and Market Rare Disease Candidates","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","year":"2020","type":"Collaboration","leadProduct":"RBCEV-based gene therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Takeda Pharmaceutical","amount2":0.90000000000000002,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.90000000000000002,"dosageForm":"","sponsorNew":"Takeda Pharmaceutical \/ Carmine Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Takeda Pha.."},{"orgOrder":0,"company":"University of Oxford","sponsor":"Evox Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Evox Therapeutics Announces Launch of Strategic Collaboration with the University of Oxford","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"Undisclosed","date":"November 2020","year":"2020","type":"Collaboration","leadProduct":"Exosome-based therapeutics","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"University of Oxford","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"University of Oxford \/ Evox Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"University.."},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"N-Lorem Foundation Partners with Ultragenyx to Bolster Development of Personalized Medicines for Patients with Ultra-Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"Undisclosed","date":"April 2021","year":"2021","type":"Collaboration","leadProduct":"Antisense oligonucleotide","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Ultragenyx Pharmaceutical","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Ultragenyx.."},{"orgOrder":0,"company":"InveniAI","sponsor":"Bristol Myers Squibb","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Relief Therapeutics and InveniAI Sign a Strategic Collaboration Agreement to Identify New Product Development Opportunities using Artificial Intelligence","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"Undisclosed","date":"November 2021","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"InveniAI","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"Dosage Strength\/Form","sponsorNew":"InveniAI \/ Bristol Myers Squibb","highestDevelopmentStatusID":"2","companyTruncated":"InveniAI \/.."},{"orgOrder":0,"company":"Cytiva","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Partners with Cytiva to Bring Individualized ASO Medicines to Patients with Nano-rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","year":"2022","type":"Partnership","leadProduct":"Antisense Oligonucleotide","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Cytiva","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Cytiva \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"Cytiva \/ n.."},{"orgOrder":0,"company":"Parexel Biotech","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Parexel and n-Lorem Foundation Partner to Support Development of New Investigational Therapies for Nano-rare Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","year":"2022","type":"Partnership","leadProduct":"Antisense Oligonucleotide","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Parexel Biotech","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Parexel Biotech \/ n-Lorem","highestDevelopmentStatusID":"2","companyTruncated":"Parexel Bi.."},{"orgOrder":0,"company":"Creyon Bio","sponsor":"DCVC Bio","pharmaFlowCategory":"D","amount":"$40.0 million","upfrontCash":"Undisclosed","newsHeadline":"Creyon Bio Launches to Engineer Safe, Effective Precision Medicines on Demand for All Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","year":"2022","type":"Series A Financing","leadProduct":"Oligonucleotide-based Medicine","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Creyon Bio","amount2":0.040000000000000001,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"Creyon Bio \/ DCVC Bio","highestDevelopmentStatusID":"2","companyTruncated":"Creyon Bio.."},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"The Conrad Prebys Foundation","pharmaFlowCategory":"D","amount":"$0.3 million","upfrontCash":"Undisclosed","newsHeadline":"The Conrad Prebys Foundation to Support the Discovery and Development of Personalized ASO Medicines for Nano-rare Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","year":"2022","type":"Funding","leadProduct":"Antisense Oligonucleotide","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"n-Lorem Foundation","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"n-Lorem Foundation \/ The Conrad Prebys Foundation","highestDevelopmentStatusID":"2","companyTruncated":"n-Lorem Fo.."},{"orgOrder":0,"company":"Octantbio","sponsor":"Catalio Capital Management","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"Octant Raises $80 Million Series B Round to Support Expansion of Next-Generation Drug Discovery Platform, Announces Collaboration with Bristol Myers Squibb and Key Appointments","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","year":"2022","type":"Series B Financing","leadProduct":"Chaperon Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Octantbio","amount2":0.080000000000000002,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Octantbio \/ Catalio Capital Management","highestDevelopmentStatusID":"2","companyTruncated":"Octantbio .."},{"orgOrder":0,"company":"KBio","sponsor":"ZabBio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"KBio Announces Strategic Partnership with ZabBio to Discover and Develop Antibody Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","year":"2022","type":"Partnership","leadProduct":"Monoclonal Antibody-based Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"KBio","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"KBio \/ ZabBio","highestDevelopmentStatusID":"2","companyTruncated":"KBio \/ Zab.."},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease to Acquire LogicBio\u00ae Therapeutics to Accelerate Growth in Genomic Medicine","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","year":"2022","type":"Acquisition","leadProduct":"AAV-based Gene Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LogicBio Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"LogicBio T.."},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease, Completes Acquisition of LogicBio\u00ae Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","year":"2022","type":"Acquisition","leadProduct":"AAV-based Gene Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"LogicBio Therapeutics","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"LogicBio Therapeutics \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"2","companyTruncated":"LogicBio T.."},{"orgOrder":0,"company":"Genomic Biopharma","sponsor":"POIC e dintorni","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genomic Biopharma and Patient Advocacy Organization POIC e Dintorni APS Partner To Offer Whole Genome Sequencing To Bring Better Treatments And Cures To Those Living With Gastrointestinal Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Partnership","leadProduct":"RNA-based Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Genomic Biopharma","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Genomic Biopharma \/ POIC e dintorni","highestDevelopmentStatusID":"2","companyTruncated":"Genomic Bi.."},{"orgOrder":0,"company":"Ethris","sponsor":"Cipla","pharmaFlowCategory":"D","amount":"$16.0 million","upfrontCash":"Undisclosed","newsHeadline":"Cipla EU to Invest EUR 15 Million In Ethris, Initiating A Strategic Collaboration","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","year":"2022","type":"Collaboration","leadProduct":"ETH45","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Ethris","amount2":0.02,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.02,"dosageForm":"Inhalation","sponsorNew":"Ethris \/ Cipla","highestDevelopmentStatusID":"2","companyTruncated":"Ethris \/ C.."},{"orgOrder":0,"company":"GenKOre","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$300.0 million","upfrontCash":"Undisclosed","newsHeadline":"GenKOre Announces Collaboration with a US-based Company on In vivo Gene-editing Therapy","therapeuticArea":"Rare Diseases and Disorders","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":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"GenKOre","amount2":0.29999999999999999,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.29999999999999999,"dosageForm":"","sponsorNew":"GenKOre \/ Undisclosed","highestDevelopmentStatusID":"2","companyTruncated":"GenKOre \/ .."},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Pfizer Inc","pharmaFlowCategory":"D","amount":"$1,350.0 million","upfrontCash":"$300.0 million","newsHeadline":"Pfizer and Beam Enter Exclusive Multi-Target Research Collaboration to Advance Novel In Vivo Base Editing Programs for a Range of Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","year":"2022","type":"Collaboration","leadProduct":"Precision Genetic Medicine","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Beam Therapeutics","amount2":1.3500000000000001,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":1.3500000000000001,"dosageForm":"","sponsorNew":"Beam Therapeutics \/ Pfizer Inc","highestDevelopmentStatusID":"2","companyTruncated":"Beam Thera.."},{"orgOrder":0,"company":"Scenic Biotech","sponsor":"NICHD","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Scenic Biotech Enters Cooperative Research & Development Agreement with the National Institutes of Health for Genetic Modifier-Based Treatment Approach for Niemann-Pick Type C Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","year":"2023","type":"Agreement","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Scenic Biotech","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Scenic Biotech \/ NICHD","highestDevelopmentStatusID":"2","companyTruncated":"Scenic Bio.."},{"orgOrder":0,"company":"Evozyne","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"$400.0 million","upfrontCash":"Undisclosed","newsHeadline":"Evozyne Announces Multi-Target Research Collaboration and License Agreement with Takeda to Develop Next-Generation Gene Therapies","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Evozyne","amount2":0.40000000000000002,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.40000000000000002,"dosageForm":"","sponsorNew":"Evozyne \/ Takeda","highestDevelopmentStatusID":"2","companyTruncated":"Evozyne \/ .."},{"orgOrder":0,"company":"OMass Therapeutics","sponsor":"British Patient Capital","pharmaFlowCategory":"D","amount":"$107.0 million","upfrontCash":"Undisclosed","newsHeadline":"OMass Therapeutics Announces Appointment of New Board Chair and Series B Extension","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","year":"2023","type":"Series B Financing","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"OMass Therapeutics","amount2":0.11,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.11,"dosageForm":"","sponsorNew":"OMass Therapeutics \/ British Patient Capital","highestDevelopmentStatusID":"2","companyTruncated":"OMass Ther.."},{"orgOrder":0,"company":"Life Edit Therapeutics","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","amount":"$1,920.0 million","upfrontCash":"Undisclosed","newsHeadline":"Novo Nordisk and Life Edit Therapeutics Establish Multi-Target Collaboration to Discover and Develop Gene Editing Therapies for Rare and Cardiometabolic Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Life Edit Therapeutics","amount2":1.9199999999999999,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":1.9199999999999999,"dosageForm":"","sponsorNew":"Life Edit Therapeutics \/ Novo Nordisk","highestDevelopmentStatusID":"2","companyTruncated":"Life Edit .."},{"orgOrder":0,"company":"CAMP4 Therapeutics","sponsor":"Fulcrum Therapeutics","pharmaFlowCategory":"D","amount":"$70.0 million","upfrontCash":"Undisclosed","newsHeadline":"Fulcrum Therapeutics Signs Exclusive Global License Agreement in Rare Hematology","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2023","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"CAMP4 Therapeutics","amount2":0.070000000000000007,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0.070000000000000007,"dosageForm":"","sponsorNew":"CAMP4 Therapeutics \/ Fulcrum Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"CAMP4 Ther.."},{"orgOrder":0,"company":"St. Jude Children\u2019s Research Hospital","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Collaborates to Support Children With Extremely Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","year":"2023","type":"Collaboration","leadProduct":"Antisense Oligonucleotide","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"St. Jude Children\u2019s Research Hospital","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"St. Jude Children\u2019s Research Hospital \/ n-Lorem Foundation","highestDevelopmentStatusID":"2","companyTruncated":"St. Jude C.."},{"orgOrder":0,"company":"Invitae","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Invitae Partners with BridgeBio Pharma to Harness Genetic Insights for the Discovery of Rare Disease Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"January 2024","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Invitae","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Invitae \/ BridgeBio Pharma","highestDevelopmentStatusID":"2","companyTruncated":"Invitae \/ .."},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Ipsen","pharmaFlowCategory":"D","amount":"$1,800.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ipsen and Skyhawk Therapeutics Announce RNA Targeting Research Collaboration in Rare Neurological Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2024","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Skyhawk Therapeutics","amount2":1.8,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":1.8,"dosageForm":"","sponsorNew":"Skyhawk Therapeutics \/ Ipsen","highestDevelopmentStatusID":"2","companyTruncated":"Skyhawk Th.."},{"orgOrder":0,"company":"BlueSphere Bio","sponsor":"National Cancer Institute","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BlueSphere Bio Establishes Strategic Collaboration with the National Cancer Institute to Advance Treatments for Rare Respiratory Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2024","year":"2024","type":"Collaboration","leadProduct":"TCR T-cell Therapy","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"BlueSphere Bio","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"BlueSphere Bio \/ National Cancer Institute","highestDevelopmentStatusID":"2","companyTruncated":"BlueSphere.."},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Astellas Announces Sponsored Research Agreement with UMass Chan Medical School","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2024","year":"2024","type":"Agreement","leadProduct":"Undisclosed","moa":"","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Discovery","graph3":"Astellas Pharma","amount2":0,"therapeuticAreaNew":"Rare Diseases and Disorders","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Rare Diseases","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Astellas Pharma \/ UMass Chan Medical School","highestDevelopmentStatusID":"2","companyTruncated":"Astellas P.."}]