Sangamo and Pfizer released positive Phase I/II data from a cohort of patients treated with a higher dose of the gene therapy SB-525 for hemophilia A, but rivals are further ahead in development.
Positive early data on Sangamo Therapeutics Inc./Pfizer Inc.'s gene therapy for hemophilia A at a higher dose cohort have put momentum behind the program, but the partners are up against rivals BioMarin Pharmaceutical Inc. and Spark Therapeutics Inc., two companies that are further ahead in the race to bring the first gene therapy for the blood disorder to the market.
Pfizer and Sangamo reported interim data April 2 from the Phase I/II Alta study, showing SB-525 was well tolerated and...
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 Commercial success remains mixed and some products have been withdrawn from the market.
