The biotech announced updated interim Phase I/II data for its gene therapy AMT-130, showing statistically significant improvement compared with an external control.
Updated interim data from uniQure N.V.’s Phase I/II study of its gene therapy AMT-130 in Huntington’s disease showed slowing of decline among treated patients compared with an external control, raising the company’s optimism that it may have a pathway to accelerated approval from the US Food and Drug Administration.
The data released 9 July show that patients receiving the high dose of AMT-130 had an 80% slowing of disease...
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 Commercial success remains mixed and some products have been withdrawn from the market.
