NEWS

The SSADH Association funded the creation of a new mouse model to be created at Boston Children’s Hospital by Alexander Rotenberg, MD PhD and Henry Lee, MPhil, PhD.  This mouse model allowed the researchers to turn on and off the SSADH gene mutation at different stages in the animal’s life to better simulate the human condition.

Once created and successfully working, the SSADH Association then funded Drs. Rotenberg and Lee, to test gene therapy solutions on the animal model.  Their work on the mouse model demonstrated that gene therapy can successfully reverse the effects of SSADH Deficiency.

In 2023, Drs. Rotenberg and Lee co-founded a company called Galibra Neuroscience.  The goal that they have for Galibra is to bring Gene Therapy to GABA-related disorders, beginning with SSADH.

Galibra announced on August 12, 2025 that they obtained the licensing rights to utilize the technology developed by Apertura Gene Therapy for the treatment of SSADH Deficiency.

This press release outlines an exciting step forward for Galibra and the SSADH community.

[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.

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.

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.