Partnership will supply material for IND-enabling non-clinical studies and a Phase 1 trial of Galibra's AAV gene therapy for a rare pediatric neurometabolic disorder, which restored survival in 100% of treated mice in preclinical studies
INDIANAPOLIS, Oct. 14, 2026 /PRNewswire/ -- Genezen, a gene therapy contract development and manufacturing organization (CDMO), and Galibra Neuroscience, a biotechnology company developing transformative therapies for rare neurological disorders, today announced a manufacturing partnership to advance Galibra's GAL005 program, an AAV-based gene therapy for succinic semialdehyde dehydrogenase (SSADH) deficiency. 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, behavioral challenges, and about half have seizures. There are no approved therapies that address the underlying cause of this disease.
After their son Sam was diagnosed in 1998, Brad and Carolyn Hoffman founded the SSADH Association, which has raised millions of dollars for research. The SSADH Association has supported a natural history study and patient-derived stem cell lines at Boston Children's Hospital, as well as the preclinical gene therapy research of Drs. Alexander Rotenberg and Henry Lee, who went on to co-found Galibra Neuroscience.
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. This second-generation, intravenously (IV) administered AAV capsid is targeted to bind human transferrin receptor 1 (hTfR1) that crosses the blood-brain barrier to deliver its payload to the central nervous system. The Galibra program has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). Galibra's preclinical data are highly encouraging with normalization of brain chemistry and disease symptom reversal in SSADH knockout mice treated with their product.
"When Sam was diagnosed, my wife Carolyn and I decided we were going to do something about it, and we have been searching for a solution for nearly three decades," said Brad Hoffman, Director at Galibra Neuroscience and Co-Founder of the SSADH Association. "We have always believed gene therapy was the answer, and the animal data are remarkable. This partnership brings us one step closer to the clinic and to the treatment our community has been waiting for."
Genezen will support this program at its Indianapolis facility. Galibra selected Genezen following an evaluation of manufacturing partners, citing the team's AAV expertise, genuine engagement with the program, and strong recommendations from the rare disease community, as well as its potential as a long-term partner across Galibra's broader pipeline beyond rare diseases.
"Brad, Carolyn, and the SSADH Association have spent nearly 30 years building the foundation for this moment, and we are honored and humbled that Galibra has trusted us to help carry it into the clinic," said Susan D'Costa, Chief Technical and Commercial Officer at Genezen. "For a community that has waited this long, manufacturing should never be the step that slows a therapy down. Our team will apply our platform AAV process and analytical methods to build a robust, well-characterized process from the start, giving Galibra a strong quality foundation for its IND and Phase 1 manufacturing. We look forward to being a long-term partner as Galibra's pipeline grows."
About Genezen
Genezen is a best-in-class gene therapy CDMO with over 12 years of experience and state-of-the-art viral vector manufacturing facilities. From concept to commercial scale, Genezen partners with innovators to deliver life-saving gene and cell therapies worldwide. Learn more at genezen.com or follow Genezen on LinkedIn.
About Galibra Neuroscience
Galibra Neuroscience, founded by Drs. Lee and Rotenberg at Boston Children's Hospital, is a biotechnology company that is developing transformative therapies for rare neurologic disorders. Through scientific innovation and close collaboration with patient communities, Galibra advances programs that address the underlying biology of devastating genetic diseases. For more information, visit www.galibraneuroscience.com.
SOURCE Genezen
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