gene therapy

Adverum Biotechnologies Inc (NASDAQ: ADVM) announced new data from the OPTIC study of ADVM-022 for wet age-related macular degeneration (wet AMD) during the American Society of Retina Specialists 2022 Annual Meeting.  New data include best-corrected visual acuity (BCVA),...

Sarepta Therapeutics Inc (NASDAQ: SRPT) shared new functional data across multiple studies from the SRP-9001 (delandistrogene moxeparvovec) program for Duchenne muscular dystrophy.  SRP-9001 is an investigational gene therapy under development in partnership with Roche...

MeiraGTx Holdings plc (NASDAQ: MGTX) announced topline data from the Phase 1/2 study MGT009 of botaretigene sparoparvovec, gene therapy for X-linked retinitis pigmentosa (XLRP). Treatment with botaretigene sparoparvovec was found to be generally safe and well-tolerated. Significant...

The FDA has placed a clinical hold on Astellas Pharma Inc's (OTC: ALPMY) FORTIS Phase 1/2 trial following a serious adverse event (SAE) of peripheral sensory neuropathy in one of the trial participants.  FORTIS trial is evaluating AT845, an investigational adeno-...

UniQure N.V. (NASDAQ: QURE) announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase 1/2 trial of AMT-130 gene therapy for Huntington's disease.  Six of the ten enrolled patients received AMT-130, and four received an...

BMO Capital initiated coverage on Beam Therapeutics Inc (NASDAQ: BEAM) with a Market Perform rating and a price target of 41. BMO writes that it appreciates the high potential of Beam's next-generation editing approach, but clinical validation is yet to be established....

CRISPR Therapeutics (NASDAQ: CRSP) presented results from its ongoing Phase 1 COBALT-LYM CTX130 (exa-cel) trial, an allogeneic CAR-T cell therapy targeting CD70 for solid tumors and certain hematologic malignancies. CTX130 was well tolerated across all dose levels. There were no cases of...

The much-awaited one-year data for the entire low-dose cohort for UniQure NV's (NASDAQ: QURE) AMT-130 gene therapy in Huntington's disease is expected at any time. The initial results from the first four patients released in December 2021 disappointed expectations as the company...

Mustang Bio Inc (NASDAQ: MBIO) announced interim data from the Phase 1/2 trial of MB-107, lentiviral gene therapy for X-linked severe combined immunodeficiency (X-SCID), also known as bubble boy disease, in newly diagnosed infants under the age of two. X-SCID is characterized by the...

Rocket Pharmaceuticals Inc (NASDAQ: RCKT) has announced topline safety and efficacy data from its Phase 2 trial of RP-L201 gene therapy in severe Leukocyte Adhesion Deficiency-I (LAD-I). Data were shared at the American Society of Gene and Cell Therapy. LAD-I is a disorder that...

Avrobio Inc (NASDAQ: AVRO) announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in infantile-onset Pompe disease.  Pompe disease is a lysosomal disorder caused by a mutation in the GAA gene. The lack of the enzyme encoded by GAA...

AVROBIO Inc (NASDAQ: AVRO) reported new interim data from the Phase 1/2 gene therapy trial of AVR-RD-04 for cystinosis. Cystinosis is a condition characterized by the accumulation of the amino acid cystine (a building block of proteins) within cells.  Excess cystine damages...

Here's a roundup of top developments in the biotech space over the last 24 hours: Stocks In Focus FDA May Authorize Pfizer-BioNTech's COVID-19 Booster For 5-11 Year Kids Soon: NYT The FDA is expected to authorize a booster shot of Pfizer Inc (NYSE: PFE)/BioNTech SE's (...

Applied Genetic Technologies Corp (NASDAQ: AGTC) reported three-month interim data from its ongoing Phase 2 Skyline trial of AGTC-501 for X-linked retinitis pigmentosa (XLRP), an inherited condition causing progressive vision loss in boys and young men.  AGTC-501 is a...

Adverum Biotechnologies Inc (NASDAQ: ADVM) announced the presentation of a new post-hoc analysis from the OPTIC study of ADVM-022 in wet age-related macular degeneration (wet AMD). Data were shared at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual...