Sarepta, Lysogene End Gene Therapy Pact for Rare Inherited Disorder

Lysogene has ended its licensing deal with Sarepta Therapeutics for its LYS-SAF302, an experimental therapy for treating a rare inherited disease of the central nervous system called mucopolysaccharidosis Type IIIA.

The termination, set to take effect on July 11, followed unsuccessful discussions between the two drugmakers regarding responsibility for global commercial supply, the companies said.

LYS-SAF302, which delivers a working copy of a gene directly to the brain to produce an enzyme called sulfamidase, is currently being evaluated in a phase 2/3 trial in 20 participants. The study’s estimated completion date is March 2022.

The FDA has granted LYS-SAF302 Orphan Drug, Fast-Track and Rare Pediatric Disease designations. The therapy has also won Orphan Drug status in Europe.