Developing Treatments for GABA-Disorders

Our vision

At Galibra Neuroscience, our mission is to lead innovation in gene therapy for GABA-related disorders.

We are committed to the translation of cutting-edge science to meet urgent patient needs.

We aim to empower lives and set new standards for excellence in personalized healthcare.

About Us


Galibra Neuroscience was founded in 2023 by a clinician and a scientist at Boston Children’s Hospital affiliated with Harvard Medical School. Our founders have over 25 years of experience in clinical care and translational research. We are directing our knowledge to improve patient lives.

Our Science

GABA (gamma-aminobutyric acid) is the major brain inhibitory neurotransmitter.

GABA keeps our neural communication in check, preventing over-excitation. GABA level in healthy brains is highly regulated (i.e., balanced).

Over 20 genes are responsible for regulating GABA signaling. Variants of these genes lead to GABA imbalance, the root cause of many rare pediatric disorders.

Because we know precisely where these genes are and what they do, replacing them (by gene replacement therapy) to achieve GABA balance is a realistic therapeutic goal.


We leverage strong partnerships with leading academic institutes and patient advocacy groups to streamline bench to bedside translation of novel disease-modifying therapies.

Our approach is RAPID:



Alexander Rotenberg, MD PhD


Joseph J. Volpe Chair and Professor of Neurology, Boston Children’s Hospital, Harvard Medical School

Henry Lee, MPhil, PhD


Senior Scientist and Program Manager, Boston Children’s Hospital, Harvard Medical School

Amber Freed

Chief Development Officer

Founder & CEO, SLC6A1 Connect

Brad Hoffman, MBA

Director of Business Development

Founder & CEO, SSADH Association

Phillip Pearl, MD

Clinical Study Advisor

William G. Lennox Chair and Professor of Neurology, Boston Children’s Hospital, Harvard Medical School

Guangping Gao, PhD

AAV Manufacturing Advisor

Penelope Booth Rockwell Professor in Biomedical Research, UMass Chan Medical School

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