Genezen, a gene therapy contract development and manufacturing organisation (CDMO), announced on Wednesday that it has entered into a manufacturing collaboration with Galibra Neuroscience, a biotechnology company developing transformative therapies for rare neurological disorders.

The collaboration is aimed at advancing Galibra's GAL005 program, an AAV-based gene therapy for succinic semialdehyde dehydrogenase (SSADH) deficiency.

Under the partnership, Genezen will produce material for IND-enabling toxicology and biodistribution studies, and manufacture cGMP clinical material for a planned Phase 1 trial.

SSADH deficiency is a rare, inherited disorder of GABA metabolism caused by mutations in the ALDH5A1 gene which encodes the SSADH enzyme. Children with the condition experience developmental delay, impaired speech, low muscle tone, behavioural challenges, and about half have seizures. There are no approved therapies that address the underlying cause of this disease.

Galibra's one-time gene therapy is designed to restore a working copy of the ALDH5A1 gene using CapX, a novel capsid developed by Apertura Gene Therapy. The Galibra programme was awarded Orphan Drug and Rare Pediatric Disease designations from the US Food and Drug Administration (FDA).