Vesigen Therapeutics Inc. has been awarded a grant by the Friedreich’s Ataxia Research Alliance (FARA) to develop a targeted genome editing therapeutic strategy for Friedreich’s ataxia.
After a long and bumpy road to approval, Reata Pharmaceuticals Inc. is to roll out the first treatment for Friedreich’s ataxia in the U.S. for patients ages 16 and older after the FDA gave its anti-inflammatory drug, Skyclarys (omaveloxolone), the green light.
Shares of Reata Pharmaceuticals Inc. tanked unexpectedly Feb. 27, a day before the U.S. FDA is set to make a decision on the NDA seeking approval of once-daily Nrf2 activator omaveloxolone for Friedreich’s ataxia, on reports that Billy Dunn, the director of the FDA’s Office of Neuroscience, was stepping down from his position.
The recent online publication of findings from the University of Southern California ataxia working group called Enigma served to fuel more interest in the simmering drug development space of Friederichs’s ataxia (FRDA), where a handful of gene therapies and other approaches, plus one promising small-molecule treatment, are in the works.
LONDON – In a sizeable seed round for a U.K. biotech, Transine Therapeutics Ltd. has raised £9.1 million (US$12.9 million) to take forward a novel method for up-regulating endogenous protein production using a naturally occurring class of long noncoding RNAs.
The brakes have now been pumped twice at Larimar Therapeutics Inc. as the FDA placed a clinical hold on its CTI-1601 Friedreich’s ataxia (FA) clinical program, and the company won’t close on a planned $95 million private placement financing. Larimar’s stock (NASDAQ:LRMR) took a battering May 26 as shares slumped 33.46% to close at $8.99.
There’s been a change of plans. Following a preliminary review of briefing materials for a type C meeting, the FDA told Reata Pharmaceuticals Inc. that a pre-NDA meeting is instead the next best step in the development of omaveloxolone (RT-408) for treating Friedreich’s ataxia (FA).
Three companies set terms for their IPOs that, if launched, will go far in maintaining the year’s already powerful momentum. Largest of the three comes from Design Therapeutics Inc., which looks to raise net proceeds of $209.1 million to fund its Friedreich’s ataxia program through IND studies and a phase I trial.
Lexeo Therapeutics Inc., a New York-based startup initially advancing clinical and near-clinical stage candidates for Friedreich’s ataxia, CLN2 and Alzheimer's diseases, has raised an $85 million series A financing round led by Longitude Capital and Omega Funds. Founded by gene therapy development veteran Ronald Crystal, the company is led by CEO Nolan Townsend, the former head of Pfizer Inc.'s rare disease efforts in North America.
Aavantibio Inc., with the help of a large check from Sarepta Therapeutics Inc., has launched with a $107 million series A to pursue gene therapies for treating rare genetic diseases. The lead program is in Friedreich’s ataxia (FA), a rare genetic disease that typically begins in childhood and causes progressive damage to the nervous system.
