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
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.
Why GALIBRA?
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:
- R - research-oriented: our data-driven approach maximizes safety and efficacy
- A - accessibility: we work closely with patient communities
- P - patient-focused: we aim to improve patient quality of life
- I - innovative: our work stems from state-of-the-art technology
- D - deep knowledge: we understand detailed disease mechanisms
Pipeline
Leadership
Alexander Rotenberg, MD PhD
Co-Founder
Joseph J. Volpe Chair and Professor of Neurology, Boston Children’s Hospital, Harvard Medical School
Henry Lee, MPhil, PhD
Co-Founder
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
