NEWS AND UPDATES
February 2026
Galibra received its third and forth funding support from two individual investors.
January 2026
Galibra was featured in the JPM RESI Conference in San Francisco, as a ‘Judges’ Pick’ recognizing ‘standout companies for exceptional science, compelling business models, and strong execution’.
Brad Hoffman was appointed Chief Financial Officer of Galibra Neuroscience.
December 2025
Galibra was featured in a showcase presentation at the New England Venture Summit 2025 in Boston.
Galibra was featured in the cover story of the Life Sciences Review magazine.
Amber Freed was featured in an exclusive interview in People.com regarding her quest to create the world’s first treatment for her son.
Galibra was invited to present updates on SLC6A1 gene therapy and participated in a panel discussion in the SLC6A1 Symposium at Atlanta, Georgia.
September 2025
Dr Phillip Pearl, Dr Alexander Rotenberg, and Dr Henry Lee were featured in a series of presentations in the International League Against Epilepsy (ILAE) Conference (Lisbon, Portugal) at a Special Interest Session titled ‘New Challenges and Progress in Treatment of Inherited Metabolic Epilepsies’.
Galibra was featured in a showcase presentation at the LSX World Congress 2025 in Boston.
Dr Phillip Pearl was featured in a recent podcast on his journey as a pediatric neurologist and recent advances in treatment for metabolic epilepsies.
Part 1: https://www.youtube.com/watch?v=yJ31k_6xNNg
Part 2: https://www.youtube.com/watch?v=scBYPashBAs
August 2025
Galibra was awarded an NIH STTR Award 1R41NS145933-01 titled ‘Developing Brain Penetrable AAV Gene Therapy for Succinic Semialdehyde Dehydrogenase Deficiency’. This project aims to package GAL005 into a translatable CapX capsid for advanced pre-clinical efficacy testing and de-risking purposes.
Press Release
Galibra Neuroscience is developing a gene therapy for rare GABA-related disorders using a blood-brain barrier-penetrating AAV capsid licensed from Apertura Gene Therapy
- Galibra is pioneering gene replacement solutions that restore gamma-aminobutyric acid (GABA) homeostasis, addressing the root mechanism behind multiple rare and debilitating childhood neurologic diseases
- Galibra has in-licensed Apertura’s TfR1 CapX AAV capsid, which binds to human transferrin receptor (hTfR1), to advance its pipeline of GABA disorder programs
[Boston MA, August 12, 2025] – Galibra obtained licensing rights to utilize Apertura’s proprietary TfR1 CapX AAV capsid to develop gene therapies targeting GABA-related disorders that include succinic semialdehyde dehydrogenase deficiency (SSADHD1,2) and SLC6A1-related disorders.
Galibra’s preclinical work at Boston Children’s Hospital indicates that SSADHD symptoms may be reversible by gene augmentation therapy. Notably, an efficacious treatment requires that the transgene — a functional copy of the defective gene — be broadly expressed throughout the brain and in critical organs such as heart and liver. Near-future translation of this gene therapy into the clinic will thus require an AAV capsid capable of widespread distribution throughout the central nervous system.
Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, developed the TfR1 CapX vector based on recent work from Dr. Ben Deverman, Senior Director of Vector Engineering at The Broad Institute of Harvard and MIT and Scientific Founder of Apertura. TfR1 CapX is an intravenously (IV) administered AAV capsid that binds to hTfR1 to cross the blood-brain barrier and effectively transduce cells throughout the central nervous system3.
Galibra identified TfR1 CapX as an excellent candidate to target critical tissues most affected in patients with GABA-related disorders. The company is highly optimistic about this novel technology’s promise to safely deliver the proprietary SSADH gene therapy payloads to desired cellular targets, resulting in minimally invasive, highly effective, and safe therapeutic options for patients with unmet needs.
About Galibra Neuroscience:
Galibra is a gene therapy company aimed to develop cure for neurologic disorders via balancing brain excitation and inhibition. Galibra is co-founded by Drs. Alexander Rotenberg and Henry Lee at Boston Children’s Hospital and Harvard Medical School, with a vision to apply cutting-edge molecular neuroscience to treat rare genetic neurologic disorders and then leverage their knowledge and experience to tackle more prevalent neurologic diseases. Please follow us on LinkedIn.
About Apertura Gene Therapy:
Apertura Gene Therapy is a biotechnology company dedicated to advancing gene therapies that address severe, unmet needs. Apertura is based in New York City, and was founded in 2021 on technology from the Broad Institute with support from Deerfield Management Company. For more information, please visit our website at www.aperturagtx.com and follow us on LinkedIn.
References:
- Henry HC Lee, Gabrielle E McGinty, Phillip L Pearl, Alexander Rotenberg. “Understanding the Molecular Mechanisms of Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD): Towards the Development of SSADH-Targeted Medicine.” J. Mol. Sci., February 26, 2022
- Henry HC Lee, Itay Tokatly Latzer, Mariarita Bertoldi, Guangping Gao, Phillip L Pearl, Mustafa Sahin, Alexander Rotenberg. “Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency.” J Inherit Metab Dis., March 20, 2024.
- Qin Huang, Ken Y. Chan, Jason Wu, Nuria R. Botticello-Romero, Qingxia Zheng, Shan Lou, Casey Keyes, Alexander Svanbergsson, Jencilin Johnston, Allan Mills, Chin-Yen Lin, Pamela P. Brauer, Gabrielle Clouse, Simon Pacouret, John W. Harvey, Thomas Beddow, Jenna K. Hurley, Isabelle G. Tobey, Megan Powell, Albert T. Chen, Andrew J. Barry, Fatma-Elzahraa Eid, Yujia A. Chan, Benjamin E. Deverman. “An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery.” Science, May 16, 2024.
Contacts:
June 2025
Dr Alexander Rotenberg is named 2025 Harrington Scholar-Innovator Award recipient by the Harrington Discovery Institute, for his project titled ‘Gene therapy for a rare inherited disease, Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD)’.
Galibra Neuroscience received its second funding support from a patient advocacy group.
May 2025
Dr Henry Lee presented our work on SSADH-targeted gene replacement therapy and hosted a scientific session ‘Translational strategies for cell and gene therapies in inherited neurotransmitter disorders’ in the American Society of Gene & Cell Therapy (ASGCT) annual meeting in New Orleans, May 13-17.
April 2025
Galibra Neuroscience presented our SSADH-targeted gene replacement therapy (GAL005) in the World Orphan Drug Congress USA in Boston, April 22-24.
March 2025
Galibra Neuroscience was invited to participate in the Investival Showcase USA Event at Miami, March 10-11.
December 2024
Galibra Neuroscience presented updates in the SLC6A1 and CURE GABA-A Scientific Meetings at Los Angeles.
Dr Henry Lee presented in the Future Benchmarks of Epilepsy Care session and latest work on SSADH at the Annual Meeting of American Epilepsy Society.
Dr Henry Lee was named one of the Young Investigators of the Rare Epilepsy Network 2024.
Amber Freed was featured in the Rare Documentary premiered at Harvard Medical School
September 2024
Construction of an inducible GABA-T mouse model has begun.
July 2024
Dr Henry Lee discussed the key elements of developing gene therapies for inherited disorders of neurotransmission including SSADH deficiency in a podcast at JIMD.
May 2024
Dr Henry Lee presented our work on SSADH deficiency in the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting.
Galibra Neuroscience obtained Option License Agreement from Boston Children’s Hospital for developing AAV-mediated gene supplement therapy for SLC6A1-related disorders.
April 2024
Dr Guangping Gao joined Galibra Neuroscience as AAV Manufacturing Consultant.
Brad Hoffman joined Galibra Neuroscience as Director of Business Development.
January 2024
Galibra Neuroscience received its first funding support from an individual investor.
December 2023
Our work on SSADH deficiency was featured in a poster highlight session in the American Epilepsy Society Annual Meeting.
Amber Freed joined Galibra Neuroscience as Chief Development Officer.
Galibra Neuroscience obtained Option License Agreement from Boston Children’s Hospital for developing AAV-mediated gene replacement therapy for SSADH deficiency.
Dr Phillip Pearl joined Galibra Neuroscience as Clinical Study Consultant.
2023
Galibra Neuroscience was founded by Drs Alexander Rotenberg and Henry Lee.
