Less than a year after the US Food and Drug Administration allowed its Phase I/II study of AT132 for X-linked myotubular myopathy (XLMTM) to resume following a months-long clinical hold over adverse events, Astellas Pharma, Inc. has hit the pause button on the gene therapy trial again due to a safety issue. The hold emphasizes the growing concern about the safety of adeno-associated viral (AAV) vector-based gene therapies in general on the eve of an FDA advisory committee on the topic.
Astellas Pauses XLMTM Gene Therapy Trial After Another Safety Scare
The day before an FDA advisory committee meeting about the safety of AAV vector-based gene therapies, Astellas reported liver damage in a patient in its trial of AT132.
More from Clinical Trials
• By
Arbele has emerged as a global leader in the Cadherin 17 area, not only in clinical progress but also for its “secret recipe” for application in T-cell engagers, its CEO tells Scrip.
• By
AstraZeneca and Daiichi’s DESTINY-Breast09 trial testing Enhertu with pertuzumab versus standard of care showed a statistically significant and meaningful PFS improvement.
• By
The IL-4/IL-13 inhibitor has been approved in the US as the first targeted therapy in over a decade for chronic itching.
• By
BriaCell announced its cell therapy Bria-IMT plus checkpoint inhibitors bested Gilead’s Trodelvy in a Phase II trial in HR+ breast cancer; a Phase III trial is enrolling.
More from R&D
• By
Start-ups from China dominated the fastest-growing share prices for mid-cap biopharma companies in Q1, while the biggest decliners were mainly US firms.
• By
While Henlius Biotech’s PD-L1-targeting antibody-drug conjugate HLX43 is struggling to catch up with Pfizer’s PF-08046054, the global frontrunner in the space, a key differentiation of the China-originated asset could lie in PD-L1-negative patients.
• By
AstraZeneca logs growth in Japan despite a big reimbursement price cut for one of its top sellers and expects 40 approvals in the country over the next six years.