After the U.S. FDA granted clearance in March 2023, sales began promptly of Acadia Pharmaceuticals Inc.’s new Rett syndrome therapy, Daybue (trofinetide), and questions during the recent J.P. Morgan Healthcare Conference in San Francisco had to do with – among other matters – sales guidance from the firm, which is hardly alone in seeking treatments for the disease. Other players include Anavex Life Sciences Corp., Neurogene Inc. and Taysha Gene Therapies Inc.
Taysha Gene Therapies Inc.’s stock (NASDAQ:TSHA) struggled Feb. 1 following the U.S. FDA’s recommendation that the company dose more patients in a double-blind, placebo-controlled study of TSHA-120 for the ultra-rare indication giant axonal neuropathy.
Astellas Pharma Inc. has invested $50 million in Taysha Gene Therapies Inc. in exchange for 15% of the company and exclusive options to in-license Taysha’s lead gene therapy candidates, TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy.
The recent start of Neurogene Inc.’s first-in-human trial testing NG-101 in ceroid lipofuscinosis type 5 (CLN5) Batten disease (BD) drew investor attention to the space, where a handful of developers vie for new therapies against the challenging indication.
A third-quarter progress report from the international advocacy group Alliance for Regenerative Medicine (ARM) has determined that the regenerative medicine and advanced therapy (RMAT) sector established a highwater mark of $15.9 billion in global financings, breaking the previous annual record of $13.5 billion that was set in 2018.
Taysha Gene Therapies Inc., a new Dallas-based company reuniting former executives of Avexis Inc. and its funders, has launched with a $30 million seed financing intended to advance a pipeline of 15 new AAV-based candidates. Its team expects to file four INDs by the end of 2021, starting with one for GM2-gangliosidosis that could move to the clinic later this year.