[{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Lonza Group","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Lonza Expands Partnership with Rocket Pharmaceuticals for Childhood Disorder Treatment","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inovio Receives Authorization from FDA To Begin Trial for INO-3107","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Vaccine","graph2":"Inovio Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Presents Positive Updates on FA and LAD-I Gene Therapy Programs at the 23rd ASGCT Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Receives Orphan Drug Designation From for DNA Medicine INO-3107 To Treat Rare Disease Recurrent Respiratory Papillomatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Vaccine","graph2":"Inovio Pharmaceuticals"},{"orgOrder":0,"company":"Oncopeptides","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Oncopeptides Starts the First Study with Melflufen Outside Multiple Myeloma and Enrolls First Patient in Phase 1\/2 AL Amyloidosis Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWEDEN","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Oncopeptides"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Presents Positive LAD-l Clinical Update and Comprehensive IMO Preclinical Review at the ESID 2020 Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Announces Dosing of First Subject in Phase 1\/2 Clinical Trial for INO-3107","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Inovio Pharmaceuticals"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Sofinnova Investments","pharmaFlowCategory":"D","amount":"$55.0 million","upfrontCash":"Undisclosed","newsHeadline":"Atsena Therapeutics Raises $55 Million Series A Financing to Advance LCA1 Gene Therapy Clinical Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atsena Therapeutics"},{"orgOrder":0,"company":"Foresee Pharmaceuticals","sponsor":"TRPharm","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Foresee Pharmaceuticals and TRPharm Announce License and Co-Development Agreement","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Foresee Pharmaceuticals"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Patient Dosed in CAN106 Phase 1b\/2 Trial for Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Announces Dosing of First Pediatric Patient in the BRILLIANCE Clinical Trial of EDIT-101 for LCA10","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","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"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Taysha Gene Therapies Announces Oral Presentations at the Upcoming 2022 IRSF Rett Syndrome Scientific Symposium and the ASCEND National Summit","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Taysha Gene Therapies"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Presents Data on SLEEK Gene Editing Technology at the American Society of Gene and Cell Therapy Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Editas Medicine"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Venthera","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Affiliate Venthera Presents Preliminary Results from Clinical Trial of VT30 (BBP-681) for Venous, Lymphatic, and Venolymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Announces Positive Results from Phase I\/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-Associated Leber Congenital Amaurosis (LCA1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atsena Therapeutics"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LEXEO Therapeutics Receives Orphan Drug Designation for LX1004 from European Commission","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lexeo Therapeutics"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Announces Clinical Data Demonstrating Proof of Concept of EDIT-101 from Phase 1\/2 BRILLIANCE Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Editas Medicine"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Opus Genetics Receives FDA Clearance of IND Application for OPGx-001, a Gene Therapy Candidate Intended for the Treatment of Rare Inherited Retinal Disease LCA5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Opus Genetics"},{"orgOrder":0,"company":"Genethon","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Genethon"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics to Present Positive Interim Encore Data from the Phase I\/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital Amaurosis (LCA1) at the 46th Annual Macula Society Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atsena Therapeutics"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Announces Positive 6-month Data from Ongoing Phase I\/II Clinical Trial of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atsena Therapeutics"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Committee for Orphan Medicinal Products Provides Positive Opinion on INOVIO's Application for Orphan Drug Designation for INO-3107","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Inovio Pharmaceuticals"},{"orgOrder":0,"company":"Innoviva","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Announces Orphan Drug Designation for INO-3107 for the Treatment of Recurrent Respiratory Papillomatosis from the European Commission","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Vaccine","graph2":"Innoviva"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"CARE Pharma Shanghai Ltd.","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge Reports Positive Preliminary Data in the Omoprubart (CAN106) Phase 1b Trial in Paroxysmal Nocturnal Hemoglobinuria (PNH) in China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme Pharma Announces Dosing of First Infant with ENPP1 Deficiency in a Phase 1b Trial of INZ-701","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Inozyme Pharma"},{"orgOrder":0,"company":"Wacker Chemie AG","sponsor":"Caeregen Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Caeregen Therapeutics and Wacker Biotech Announce Collaboration Agreement to Produce the Regenerative Medicine Clinical Candidate CTR-107 (Noregen\u2122) for Retinal Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Wacker Chemie AG"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme Pharma Announces Updates on Global Development Strategy of INZ-701 for the Treatment of ENPP1 Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Inozyme Pharma"},{"orgOrder":0,"company":"MyoPax","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MyoPax Receives FDA Orphan Drug Designation for its Innovative Regenerative Cell Therapy for Exstrophy-Epispadias Complex","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"GERMANY","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"MyoPax"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"BofA Securities","pharmaFlowCategory":"D","amount":"$60.0 million","upfrontCash":"Undisclosed","newsHeadline":"Inozyme Pharma, Inc. Announces Pricing of Public Offering of Common Stock","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Inozyme Pharma"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"BofA Securities","pharmaFlowCategory":"D","amount":"$64.5 million","upfrontCash":"Undisclosed","newsHeadline":"Inozyme Pharma, Inc. Announces Closing of Public Offering of Common Stock","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Inozyme Pharma"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"Benitec Biopharma Inc. Announces Pricing of $30 Million Underwritten Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Benitec Biopharma"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Opus Genetics Announces First Patient Dosed in Phase 1\/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare Inherited Retinal Disease LCA5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Opus Genetics"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Announces U.S. FDA Breakthrough Therapy Designation Granted for INO-3107 for the Treatment of Recurrent Respiratory Papillomatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Vaccine","graph2":"Inovio Pharmaceuticals"},{"orgOrder":0,"company":"Viralgen","sponsor":"Elpida","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Viralgen & Elpida Partner for SPG50 & CMT4J Trials","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SPAIN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Viralgen"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces FDA Acceptance of Biologics License Application with Priority Review for RP-L201 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Receives FDA Feedback that Data from Completed Phase 1\/2 Trial of INO-3107 Can Be Used to Submit a BLA Under Accelerated Approval Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Vaccine","graph2":"Inovio Pharmaceuticals"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atsena Therapeutics"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
ATSN-101, gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101.
INO-3107 is clinical-stage DNA medicine product candidate being developed as a potential treatment for RRP. it is designed to elicit a targeted T cell response against HPV-6 and HPV-11, the HPV types responsible for causing RRP among other HPV-related diseases.
RP-L201 (marnetegragene autotemcel), a lentiviral vector (LV)-based investigational gene therapy, which is investigated for the treatment of Severe Leukocyte Adhesion Deficiency-I.
Viralgen Vector Core and Elpida Therapeutics partner to manufacture adeno-associated vector serotype 9 (AAV9) based therapies, including MELPIDA, for anticipated Spastic Paraplegia 50 (SPG50) and Charcot-Marie-Tooth disease type 4J (CMT4J) clinical trials.
OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).
INO-3107 is clinical-stage DNA medicine product candidate being developed as a potential treatment for RRP. INO-3107 is designed to elicit a targeted T cell response against HPV-6 and HPV-11, the HPV types responsible for causing RRP among other HPV-related diseases.
The company intends to use the net proceeds to support the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB-301 for the treatment of dysphagia associated with oculopharyngeal muscular dystrophy.
The company intends to use the net proceeds to fund advancement of its INZ-701 (rhENPP1-Fc) clinical program, including its ongoing Phase 1/2 trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency, its ongoing Phase 1b trial of INZ-701 for infants with ENPP1 Deficiency.
The net proceeds will fund advancement of company's INZ-701 (rhENPP1-Fc) clinical program, including its ongoing Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency.
INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton.
Market Place
Upload your Marketing & Sales content on your company Virtual Booth, click HERE
