- Genezen has partnered with Galibra Neuroscience to manufacture GAL005, an adeno-associated virus (AAV)-based gene therapy candidate for succinic semialdehyde dehydrogenase (SSADH) deficiency.
- Genezen will produce material for IND-enabling toxicology and biodistribution studies and manufacture current Good Manufacturing Practice (cGMP) clinical material for a planned Phase 1 trial.
Genezen, a gene therapy contract development and manufacturing organization (CDMO), has entered into a manufacturing partnership with Galibra Neuroscience to advance GAL005, an investigational gene therapy for SSADH deficiency, a rare inherited neurological disorder. Genezen will support the program at its Indianapolis facility, manufacturing material for preclinical studies and clinical supply for a planned Phase 1 trial.
GAL005 is designed to deliver a working copy of the ALDH5A1 gene using CapX, an adeno-associated virus (AAV) capsid developed by Apertura Gene Therapy. The intravenously administered capsid targets human transferrin receptor 1 to cross the blood-brain barrier and deliver its genetic payload to the central nervous system. Galibra’s program has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration.
SSADH deficiency is caused by mutations in the ALDH5A1 gene, which encodes the SSADH enzyme. The condition can cause developmental delays, impaired speech, low muscle tone, behavioral challenges, and seizures. There are currently no approved therapies that address the underlying cause of the disease. Galibra has reported that preclinical studies in SSADH knockout mice showed normalization of brain chemistry and reversal of disease symptoms.
Galibra selected Genezen following an evaluation of manufacturing partners, citing its AAV expertise, engagement with the program, recommendations from the rare disease community, and potential to support its broader pipeline. Genezen will apply its AAV process and analytical methods to establish a characterized manufacturing process for the program’s IND and Phase 1 requirements.
“The partnership brings the program closer to clinical development. The association, founded by Hoffman and his wife Carolyn after their son was diagnosed in 1998, has supported research into the condition, including a natural history study, patient-derived stem cell lines, and preclinical gene therapy work.”
Brad Hoffman, director at Galibra Neuroscience and co-founder of the SSADH Association
