Privately held GeneTx Biotherapeutics LLC and development partner Ultragenyx Pharmaceuticals (NASDAQ:RARE) announce that GeneTx has filed an IND with the FDA seeking signoff for a Phase 1/2 clinical trial evaluating GTX-102 in patients with a rare inherited nervous system disorder called Angelman syndrome. Unless something unexpected happens, enrollment should begin in H1.
The companies are co-developing the antisense oligonucleotide under an August 2019 agreement. Under the terms of the deal, RARE has an option to acquire GeneTx any time prior to 30 days after the FDA accepts the IND.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.