gene therapy

Regenxbio Inc (NASDAQ: RGNX) has announced a safety update and additional positive interim data from its ongoing Phase 1/2 trial of RGX-121 to treat patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. The data will be...

Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) has announced longer-term data from the Glycogen Storage Disease Type Ia (GSDIa) and Ornithine Transcarbamylase (OTC) Deficiency Phase 1/2 studies. Data were presented at the American Society of Gene & Cell Therapy (ASGCT) 24th...

Biogen Inc (NASDAQ: BIIB) has announced topline results from the Phase 2/3 XIRIUS study evaluating cotoretigene toliparvovec (BIIB112), a gene therapy being investigated as a one-time therapy for patients with X-linked retinitis pigmentosa (XLRP). XLRP is a rare,...

Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced positive data from its Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) gene therapy programs. Data were presented at the American Society of Gene and Cell Therapy Annual...

Sio Gene Therapies Inc (NASDAQ: SIOX) has announced new biomarker data from Phase 1/2 study of AXO-AAV-GM1, an adeno-associated viral vector (AAV)9-based gene therapy candidate for GM1 gangliosidosis. This new data will be presented at the American Society of Gene...

Three years after receiving gene therapy from Orchard Therapeutics (NASDAQ: ORTX) in an early-stage clinical trial, 100% of patients with an inherited disease that damages the immune system were still alive. Data from a study that used Orchard’s gene therapy OTL-101 to treat...

Precision BioSciences Inc (NASDAQ: DTIL) has announced new in vivo data using ARCUS genome editing platform resulting from a preclinical research collaboration with investigators at the University of Pennsylvania. Data were presented at the 2021 American Society of Genetic...

Applied Genetic Technologies Corporation (NASDAQ: AGTC) has announced additional data from the ongoing X-linked retinitis pigmentosa (XLRP) gene therapy Phase 1/2 trial, including 12-month data from Groups 5 & 6 and 24-month data from two of three Group 4 patients. Groups 5...

Lineage Cell Therapeutics Inc (NYSE: LCTX) has updated interim results from its ongoing Phase 1/2a study of its lead product candidate, OpRegen, for dry age-related macular degeneration (AMD) with geographic atrophy (GA). OpRegen is an investigational cell therapy consisting of...

Avrobio Inc (NASDAQ: AVRO) has provided an update on its regulatory plans for AVR-RD-01, its lentiviral gene therapy for Fabry disease, currently in FAB-GT Phase 2 trial. In March, the FDA granted full approval to Sanofi's SA (NASDAQ: SNY) Fabrazyme (agalsidase...

Adverum Biotechnologies Inc (NASDAQ: ADVM) has announced new long-term data from the OPTIC Phase 1 trial of ADVM-022 single intravitreal (IVT) injection gene therapy in patients with neovascular or wet age-related macular degeneration (wet AMD). Safety and efficacy data from...

Adverum Biotechnologies Inc (NASDAQ: ADVM) stock has cratered in reaction to a Suspected Unexpected Serious Adverse Reaction of hypotony (clinically-relevant decrease in ocular pressure).  The event was reported in the INFINITY Phase 2 trial evaluating ADVM-022...

In a financial notice posted earlier today, Astellas Pharma Inc (OTCMKTS: ALPMY) said it was taking an impairment loss of ¥58.8 billion ($540 million) because of last year’s FDA clinical hold on Audentes’ AT132, gene therapy X-linked myotubular myopathy (XLMTM), a rare...

CANbridge Pharmaceuticals Inc has entered into a strategic collaboration and licensing agreement with LogicBio Therapeutics Inc (NASDAQ: LOGC) to develop, manufacture and commercialize gene therapy candidates for treatments for Fabry and Pompe diseases. The candidates...

Today FDA has removed the clinical hold on one more gene therapy study, this time for Voyager Therapeutics Inc's (NASDAQ: VYGR) investigational new drug application for VY-HTT01, a gene therapy candidate for Huntington's disease (HD). The agency has confirmed that...