Astellas’ $3 billion bet on Audentes Therapeutics has hit yet another setback. Months after getting a clinical hold lifted, Astellas has voluntarily paused a gene therapy clinical trial after a patient suffered liver problems at the lower, supposedly safer dose.
Going into 2020, Astellas planned to file for approval of AT132 in a rare neuromuscular disease by the end of the year. Instead, three deaths linked to liver failure among the 17 patients who received the high dose of the AAV8 vector gene therapy led to a clinical hold. The FDA lifted the hold in December after Astellas dropped the