MedCity News February 1, 2023
Frank Vinluan

Taysha Gene Therapies has encouraging data from an open-label, Phase 1/2 study in the rare disease giant axonal neuropathy. It might not be enough. The FDA recommended the biotech conduct a randomized and placebo-controlled study—a challenge in any ultra-rare disease.

One challenge of developing a drug for a disease with no approved therapies is that there isn’t an established clinical trial roadmap to follow. Taysha Gene Therapies hoped the trial design for its gene therapy for a rare neuromuscular disease would put it on the path to a regulatory submission. FDA questions about the study mean that the path forward is almost certainly longer, and might not even be possible.

After meeting with FDA to discuss data from a small,...

Today's Sponsors

LEK
ZeOmega

Today's Sponsor

LEK

 
Topics: Biotechnology, Clinical Trials, FDA, Govt Agencies, Pharma / Biotech, Trends
Gilead bets on Xilio cancer drug as biotech restructures
Pfizer shifts creative to Publicis in latest marketing move
Boundless Bio’s ‘BOLD’ IPO Reels In $100M for a New Kind of Cancer Drug
Joseph La Barge On The Challenges Of Advancing Gene Therapy, Finding The Right Support
Exact Sciences' posts positive data for its capsule-on-a-string test for esophageal cancer

Share This Article