Sarepta Licenses Up to Three CNS-Targeting Gene Therapies from Lacerta

Sarepta Therapeutics will shell out potentially more than $38 million for rights to up to three preclinical CNS-targeting gene therapy candidates from Lacerta Therapeutics, in a broadening of the buyer’s pipeline of rare neuromuscular disease treatments beyond Duchenne muscular dystrophy (DMD).

Through a License, Development and Option Agreement inked by the companies, Sarepta is licensing exclusive rights to a Lacerta treatment for Pompe disease, as well as options to license two additional Lacerta candidates.

While the indications of those candidates were undisclosed by Sarepta in an announcement and a regulatory filing yesterday, Lacerta says its pipeline includes additional gene therapies for Sanfilippo Syndrome Type B; Aromatic L-amino acid decarboxylase deficiency; Neurodegenerative Proteinopathies; Spinocerebellar Ataxias (SCA); and Glioblastoma.

Lacerta has identified the Sanfilippo Syndrome Type B and SCA programs, along with the Pompe disease program, as its three lead programs.   

For the Pompe disease treatment ...