Gaba Therapeutics is focused on developing innovative therapies that enhance the body's natural production of neurosteroids to address various central nervous system (CNS) disorders. Their lead product, GRX-917, works by inducing the production of endogenous neurosteroids, such as allopregnanolone, while also inhibiting NLRP3 inflammation. This dual mechanism of action is designed to provide therapeutic benefits for a wide range of neuropsychiatric, neurological, and autoimmune disorders, with GRX-917 set to enter Phase 2/3 registration studies for anxiety in 2025. The company emphasizes the importance of neurosteroids in normal brain function, highlighting their roles in inducing anxiolytic, antidepressant, anticonvulsant, and analgesic effects.

The scientific approach of Gaba Therapeutics is to leverage the brain's intrinsic ability to correct imbalances associated with CNS disorders, rather than relying on synthetic drugs that target specific receptors. By enhancing the natural production of neurosteroids, the company aims to restore normal neurosteroid levels, which can alleviate symptoms and potentially modify disease states in various conditions, including anxiety, depression, and neurodegenerative diseases. The therapeutic strategy also aims to minimize side effects by preserving the brain's precise control over neurosteroid production, utilizing the TSPO receptor to increase local synthesis from cholesterol, thereby promoting neuroregeneration and neuroprotection.

United States

GABA Therapeutics, Inc is a clinical-stage biotechnology company developing novel therapies for anxiety, depression, and chronic pain. Their lead drug candidate, GRX-917, is a non-addictive treatment with minimal side effects, offering comparable efficacy to Xanax. The company has received approval to begin phase I clinical trials for GRX-917 and is focused on advancing innovative treatments for mental health disorders.

Gaba Therapeutics

Company Details