The company has initiated the first pivotal double-blind gene therapy trial in DMD in the US in a tight race with Pfizer.
Sarepta Therapeutics, Inc. appears to have a lead in the US over Pfizer Inc. in the race to bring the first gene therapy to market for Duchenne Muscular Dystrophy (DMD). Sarepta announced the initiation of the pivotal EMBARK trial on 4 October, testing its investigational gene therapy SRP-9001 in the US, Europe and Asia.
Sarepta and its ex-US partner Roche Holding AG have been in a close race with Pfizer, which has already...
More than 100 biopharma executives and experts told Scrip their predictions for therapeutic area advances in the coming year. The recent commercial success of GLP-1s in diabetes and obesity and their potential in further disease areas fuelled excitement around the metabolic space. Expectations were also positive in neurology following the launch of Leqembi for Alzheimer’s disease in 2023, while the multiple opportunities to improve cancer treatment kept oncology top of the pile overall.
In this week's podcast edition of Five Must-Know Things: Merck & Co. steps into CD19 bispecific space; gene therapy patients rise, but slowly; Madrigal’s Rezdiffra plans; Korean biopharma financing recovering?; and approvals to watch out for in Q3.
Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area.
After 10 years of promised investment following its Nobel Prize for iPS cell research, Japan is cautiously narrowing regulations around the conditional approval of cell therapies and cutting some reimbursement prices. Commercial success remains mixed and some products have been withdrawn from the market.
