Coave Therapeutics Preparing for Retinitis Pigmentosa Gene Therapy Registration Trial
In an ongoing Phase I/II trial, the gene therapy improved visual function in patients with early stages of the rare, inherited visual disorder.
Anew Medical Expecting to Access $54M Following SPAC Merger
The company is hoping the capital will help it advance a pipeline of gene therapies for various neurodegenerative diseases.
In Brief This Week: Clarity Pharmaceuticals, Regenxbio, Rocket Pharmaceuticals, Eisai, AstraZeneca
News items for the week of May 22, 2023.
FDA Delays Decision for Sarepta Therapeutics Duchenne Muscular Dystrophy Gene Therapy
According to Sarepta, the federal agency has indicated it may grant accelerated approval to SRP-9001 as a treatment for a subgroup of patients based on age.
PTC Therapeutics Cuts Multiple Gene Therapy Programs
The company, which focuses on rare disease gene therapies and small molecule treatments, also said its CFO will leave the firm.