Pfizer is forging ahead with a Phase III trial for its DMD therapy, even though Phase Ib data raised safety worries. Gene therapy rival Sarepta’s shares were up by 17% on the news.
Pfizer Inc. has allowed a peek into its Phase Ib clinical data on PF-06939926, its investigational gene therapy for Duchenne muscular dystrophy (DMD), and while it does show some signs of efficacy, safety is a concern.
The primary endpoint of the ongoing Phase Ib study is to assess the safety and tolerability of the gene therapy,...
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 Commercial success remains mixed and some products have been withdrawn from the market.
