gene therapy

Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced updated data from the Phase 1 trial evaluating a single intravenous infusion of RP-A501, an investigational gene therapy, for Danon Disease.  Danon disease is characterized by weakening of the heart muscle, weakening of...

Setbacks have resulted in delays across all three of Pfizer Inc’s (NYSE: PFE) gene therapy programs, the Company revealed in its Q3 conference call. Pfizer slammed breaks on its Sangamo-partnered Phase 3 hemophilia A study after some patients reported high Factor VIII levels,...

Bluebird Bio Inc (NASDAQ: BLUE) will withdraw the regulatory marketing authorization for Skysona (elivaldogene autotemcel) in the European Union. The update comes as a part of the company's winding down of operations there. Bluebird will also withdraw marketing authorization of...

Selecta Biosciences Inc (NASDAQ: SELB) has announced a strategic licensing agreement to advance a next-generation IgG protease.  The partnership leverages Genovis' immunoglobulin G (IgG) protease, IdeXork (Xork), and Selecta's ImmTOR platform to enable the dosing of...

Sio Gene Therapies Inc (NASDAQ: SIOX) has announced interim data from its Phase 1/2 study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for GM1 gangliosidosis. GM1 gangliosidosis is a genetic disorder that progressively destroys nerve cells in...

Voyager Therapeutics Inc (NASDAQ: VYGR) has announced the presentation of preclinical data of GBA1 gene replacement therapy. The data demonstrated that a single intravenous (IV) dose of GBA1 gene replacement therapy achieved widespread distribution in the central nervous system (CNS...

Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced interim data updates from seven patients from the RP-L201 Phase 1/2 trial for Leukocyte Adhesion Deficiency-I (LAD-I). Data were shared at the Annual Congress of the European Society of Gene & Cell Therapy (ESGCT). RP-L201...

Sarepta Therapeutics Inc (NASDAQ: SRPT) shared new analyses and functional data from its SRP-9001 development program and the details of Study SRP-9001-301 (EMBARK) Phase 3 trial of SRP-9001 for Duchenne muscular dystrophy.  SRP-9001, being developed in partnership with ...

Regenxbio Inc (NASDAQ: RGNX) announced initial data from the Phase 2 ALTITUDE trial of RGX-314 for diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME).  The data were presented at the American Society of Retina Specialists Annual Meeting. RGX-314...

The FDA has signed off 4D Molecular Therapeutics' (NASDAQ: FDMT) Investigational New Drug Application (IND) for 4D-150 for wet age-related macular degeneration (wet AMD).  The active IND enables the initiation of a 4D-150 Phase 1/2 clinical trial, expected before year-end....

Adverum Biotechnologies Inc (NASDAQ: ADVM) announced new long-term data from the OPTIC trial of ADVM-022 gene therapy for neovascular or wet age-related macular degeneration (wet AMD). Data were shared at the Retina Society's Annual Scientific Meeting. The 2 x 10^11 vg/eye dose...

Amicus Therapeutics Inc (NASDAQ: FOLD) has agreed to spin off its gene therapy business to Sciences Acquisition Corp IV (NASDAQ: ARYD) and launch genetic medicine company Caritas Therapeutics Inc. ARYA Sciences Acquisition Corp IV is SPAC, sponsored by Perceptive...

After escalating the dose in its pediatric gene therapy trial, Applied Genetic Technologies Corporation (NASDAQ: AGTC) has run into safety problems.  The Company recently enrolled six pediatric achromatopsia (ACHM) with CNGB3 gene mutation and five pediatric ACHM patients with CNGA3...

Verve Therapeutics Inc (NASDAQ: VERV) has reported new preclinical data from 36 non-human primates (NHPs) with its lead candidate, VERVE-101. VERVE-101 is a potential single-course gene-editing treatment for atherosclerotic cardiovascular disease (ASCVD).  The data will be...

Solid Biosciences Inc (NASDAQ: SLDB) reports 1.5-year functional data and patient-reported outcome measures in the ongoing IGNITE DMD Phase 1/2 trial of SGT-001.  SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy for Duchenne muscular...