Genezen and Galibra Neuroscience have entered a manufacturing partnership to prepare Galibra’s experimental gene therapy for succinic semialdehyde dehydrogenase deficiency for a planned first human trial.
Genezen will produce material for toxicology and biodistribution studies supporting an Investigational New Drug application, as well as clinical material for a planned Phase 1 trial. Manufacturing will take place at Genezen’s Indianapolis facility.
The therapy, GAL005, targets a rare inherited disorder caused by mutations in the ALDH5A1 gene, which encodes the SSADH enzyme. The condition disrupts GABA metabolism and can cause developmental delays, impaired speech, low muscle tone and behavioral difficulties. About half of affected children experience seizures.
There are no approved treatments addressing the disease’s underlying cause, according to the companies.
GAL005 is designed to deliver a working copy of ALDH5A1 through a single intravenous administration. It uses an adeno-associated virus capsid developed by Apertura Gene Therapy that targets human transferrin receptor 1 to deliver genetic material across the blood-brain barrier into the central nervous system.
Galibra reported normalized brain chemistry, reversal of disease symptoms and survival in all treated mice in preclinical studies. Those findings have not established safety or efficacy in humans.
The program has received FDA Orphan Drug and Rare Pediatric Disease designations.
Its development builds on research supported by the SSADH Association, founded by Brad and Carolyn Hoffman after their son’s diagnosis in 1998. Association-backed researchers Alexander Rotenberg and Henry Lee later co-founded Galibra.