Galibra Neuroscience
Restoring Balance to the Brain’s Most Fragile Chemistry

Dr. Alexander Rotenberg, Co-Founder and Dr. Henry Lee, Co-Founder

Founded by Drs. Henry Lee and Alexander Rotenberg, both leading physician-scientists at Boston Children’s Hospital and Harvard Medical School, Galibra is addressing both problems at once by targeting disorders in which the defective genes are already known. Specifically Galibra aims to restore genes in disorders where mutations interfere with the cellular management of GABA, the brain’s main inhibitory signal, by developing a gene-replacement platform capable of safely delivering those corrective genes into the brain.

Galibra’s therapies seek to normalize neural function by replacing the missing or faulty genes. To do so, the team designed a series of constructs (DNA that will replace the missing genes in patients), and licensed a viral vector that can pass through the blood–brain barrier after intravenous injection. Their unique platform ensures that the replacement genes are expressed only in the right neurons and in the right amounts, to mimic a healthy brain.

Our approach is to replace the genes that are abnormal in these patients for purposes of actually normalizing their physiology, as opposed to suppressing symptoms,” says Dr. Lee, whose calm clarity reflects both a scientist’s precision and compassion. “That is the big picture, and it is what drives everything we do.”

A Mission Born from Urgency

The story of Galibra began not in a boardroom but in a hospital corridor. Over two decades ago, Brad and Carolyn Hoffman, parents of a child affected by SSADH Deficiency, found themselves in a medical maze with no exit. They built patient advocacy networks, funded early studies, and created the infrastructure—biorepositories, natural history data, animal models, and stem-cell research—that would one day make true therapy development possible. Their path intersected with Drs. Lee and Rotenberg, who were studying the molecular mechanisms underlying these very disorders. The collaboration that followed was as organic as it was powerful, a merging of parental tenacity and scientific ingenuity.

Following on Brad and Carolyn’s groundwork, and with assistance from Alice McConnel, another parent of children with SSADH Deficiency, Lee and Rotenberg founded Galibra Neuroscience to pursue what once seemed impossible: a cure for SSADH deficiency and related conditions. “Brad and Carolyn brought not only personal urgency but also twenty-five years of research, which included natural history studies, biorepositories, and stem-cell research, and gave us an unparalleled foundation,” says Dr. Rotenberg.

Once founded, Galibra looked to expand their team. This is where they met Amber Freed, a mother of a child with SLC6A1-NDD, who joined their team. Amber, like many parents of children with recently-described genetic disease worked tirelessly to raise awareness and funded research to eventually treat the underlying genetic problem in SLC6A1-NDD. “I learned quickly that to treat my child’s disease, I need scientists in addition to doctors.” Rotenberg and Lee then added SLC6A1-NDD to their list of projects aimed to restore GABA balance and with it, the lives of patients and families.

Their early research in animal models proved highly successful. By introducing a healthy gene to replace the faulty one, they demonstrated that the two disorders, previously considered irreversible, were rescuable. Mice that once displayed the neurological hallmarks of SSADH Deficiency began to recover normal function. Mice with abnormal electrical brain activity (EEG) that characterize SLC6A1-NDD had their brain waves normalized. These discoveries validated decades of patient-led advocacy and confirmed that the root cause of certain GABA disorders could indeed be corrected.

What distinguishes Galibra from others in the gene therapy field is not only what they are fixing but how. While traditional approaches rely on flooding the brain with a replacement gene, Galibra’s therapy aims for precision, ensuring that expression occurs only where needed and at the right intensity.

This principle of restoring balance without disruption mirrors the biology of GABA itself, the neurotransmitter responsible for maintaining inhibitory tone in the brain. Too little GABA activity can lead to seizures and developmental delays; too much can dull neural communication. The key is equilibrium. Galibra’s therapy becomes, in essence, a molecular act of balance. The company achieves this through a sophisticated understanding of gene regulation.

“Every cell in the body carries the same DNA,” says Dr. Lee, “but different cell types express different sets of genes depending on their role. We use those natural promoters to ensure our therapy mimics the body’s normal expression pattern.” With the use of natural promoters, when a Galibra therapy is administered, neurons that need the missing gene express it naturally, while others remain silent, a design that prevents the risks associated with overcorrection.

The Science behind the Hope

Behind Galibra’s elegant science is an equally thoughtful network of collaborators. The company partners with Apertura Gene Therapy, whose advanced capsid technology allows vectors to cross the blood–brain barrier, one of the greatest obstacles in neurological medicine. It also benefits from the guidance of Dr. Guangping Gao, a Professor at UMass Chan Medical School who plays a central role in the discovery and characterization of adeno-associated virus (AAV) revitalizing the gene therapy filed in the last decade, and Dr. Phillip Pearl, Chief of Epilepsy and Clinical Neurophysiology at Boston Children’s Hospital, who has characterized SSADH Deficiency for more than a quarter century. This collective expertise provides Galibra with both the scientific depth and the clinical foresight necessary to translate bench discoveries into bedside realities.

“Our work is preclinical for now,” says Dr. Lee, “but we have confirmed that our construct works not only in animals but also in human stem-cell models. That is a major step forward, and we are cautiously optimistic about what is next.” The company plans to move toward first-in-human clinical trials within the next two years, pending regulatory and funding milestones. Each stage, the founders insist, will be governed by the same patience and precision that have defined their research from the start.

Patients as Partners, Not Subjects

From its inception, Galibra has been built on the principle that patients and their families are not passive participants; they are partners. The same families who helped seed early research now shape trial design, recruitment, and outreach. This collaboration creates a crucial advantage: when Galibra’s therapies are ready for clinical testing, there will already be a community of informed, engaged families prepared to participate.

“We know that we have a tangible patient population, with whom we have a close relationship, who are eager to enter into clinical trials” notes Dr. Lee. “That should accelerate some of those clinical translations when we have a drug ready and we also have the patients ready.” In a field where recruitment can delay trials for years, that readiness is invaluable. More than logistics, it reflects trust that has been earned through transparency, communication, and shared purpose.

Creating a Sustainable Ecosystem

Galibra’s leadership team is deliberately lean. Alongside its co-founders, it includes leading virologists, neurologists, and advocates. Each brings a distinct perspective: scientific, clinical, and deeply human. The founders’ ongoing roles at Boston Children’s Hospital allow them to witness firsthand the impact of the diseases they aim to cure.

Lee and Rotenberg’s roles as active scientists and physician give Galibra a unique advantage. They understand the full journey of a patient, from diagnosis to therapy design to clinical trial, and they bring that awareness into every experiment, scientific decision, and ethical consideration.

The company envisions a model in which modern pricing, reimbursement, and regulatory frameworks make curative gene therapies viable for rare diseases, a feat once thought financially untenable. Their long-term vision is not simply to pioneer the science but to create a sustainable ecosystem in which that science can thrive.

Toward a New Horizon

The company’s next wave of projects extends beyond rare diseases. Galibra is already developing broader gene therapies for drug-resistant epilepsy, which affects nearly a third of patients with epilepsy worldwide. If successful, these therapies can redefine the standard of care for millions, bridging the gap between rare genetic correction and mainstream neurology.

The next 18 to 24 months will focus on moving from preclinical validation to human trials, deepening partnerships with institutions and regulators, and expanding the pipeline to include additional GABA-related disorders.

“The ultimate goal is to build a platform that can be adapted to other diseases that can be treated by restoring GABA balance. Once we demonstrate that this works in one, we can extend it to others, since many disorders share a common biology but needing a tailored solution,” says Dr. Lee.

For the families who have waited generations for progress, this shift represents more than a scientific milestone. It is a restoration of faith in science, perseverance, and the idea that collaboration between patients and researchers can indeed change the course of disease.

“Science can be slow,” reflects Dr. Rotenberg, “but when you bring together people who live with the problem and those who have the tools to solve it, things start to move. That is where real progress happens.”