NEWS

Galibra Neuroscience has been awarded $500,000 through the U.S. Department of Health and Human Services. This grant through the Small Business Technology Transfer (STTR) program is to encourage research and development, along with the transfer of technology between small businesses and non-profit research institutions.

The award has been directed to fund Galibra’s clinical research related to neurological disorders in conjunction with Boston Children’s Hospital.  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 like SSADH Deficiency.

Drs. Rotenberg and Lee the founders of Galibra have proven using the mouse model that they can successfully reverse the effects of SSADH Deficiency.

Additionally, Wardiya Afshar-Saber, PhD., at BCH working on the SSADH human iPSC derived neurons in vitro is able to mimicking the gene therapy mouse model results.

These results will be combined to provide the foundation for the remaining steps of the Preclinical Research phase.  This is a critical step forward towards laying the ground work required by the US Food and Drug Administration (FDA) for drug approval.

Yet to be completed are the Investigational New Drug (IND) application which provides the FDA with data from preclinical research, as well as information about how the drug is made and how they plan to execute the clinical trials.

Also part of the IND meeting is a study plan, called a protocol, which includes details around:

• Participants (How many and who qualifies) 
• Duration (How long each phase and the overall study will last)
• Method of caparison (Whether there will be a control group to compare the data against)
• Assessment (What will be measured and how will it be assessed)
• Analysis (How the data will be analyzed or interpreted)

This grant will be helpful for Drs. Rotenberg and Lee to leverage their research from Boston Children’s Hospital and position Galibra to complete the IND meeting with the FDA. After the IND meeting is complete, they will get guidance from the FDA on the next steps required before the human gene therapy trial may be approved to begin.