[{"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"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
GNT-0003 is being developing a gene therapy treatment to treat patients with severe forms of CN syndrome by restoring expression of the UGT1A1 enzyme in the patient’s liver.
OPGx-001 is an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5gene to retinal photoreceptors. Preclinical studies in in vitro and in vivo models of LCA5 have provided support for the safety and efficacy of OPGx-001.
EDIT-101 is a CRISPR/Cas9-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), by deleting the IVS26 CEP290 mutant allele.
LX1004 is an adeno-associated virus based gene therapy for the treatment of CLN2 Batten disease. In Phase 1/2 clinical trial, LX1004 demonstrated a favorable safety profile and showed statistically significant reduction in rate of decline compared to natural history studies.
Data demonstrated that subretinal delivery ATSN-101 (SAR439483) was well tolerated and patients treated with highest dose clinically meaningful improvements in vision, as measured by full-field stimulus testing and multi-luminance mobility testing.
The data presented are preliminary results from an open-label, dose-finding study of VT30 (BBP-681) topical gel in patients with cutaneous VM/LM/VLM lesions associated with mutations that result in dysregulated PI3K activation.
EDIT-101 program for the treatment for Leber Congenital Amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder, demonstrating a favorable immunogenicity profile.
TSHA 102 is constructed from a neuronal specific promoter, MeP426, coupled with the miniMECP2 transgene, a truncated version of MECP2, and miRNA-Responsive Auto-Regulatory Element, or miRARE, our novel miRNA target panel, packaged in self-complementary AAV9.
EDIT-101 is a CRISPR/Cas9-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder.
CAN106 is a novel, long-acting recombinant human monoclonal antibody that binds to and neutralizes C5, a key component of the complement system for treatment of paroxysmal nocturnal hemoglobinuria.