Gain Therapeutics to Present Parkinson’s Drug Data at May 22 GBA1 Meeting
Gain Therapeutics will present an update on its Parkinson’s drug GT-02287 at the 3rd International GBA1 Meeting in Phoenix on May 22, 2026. Initial Phase 1b data showed CNS target engagement, baseline glucosylsphingosine reduction and stabilized MDS-UPDRS scores.
1. Conference Presentation Details
Gain Therapeutics will deliver an oral presentation titled “An update on the development of the GCase modulator GT-02287 for Parkinson’s disease” on May 22, 2026 at 4:15 PM MST during the 3rd International GBA1 Meeting in Phoenix, AZ. Chief Medical Officer Jonas Hannestad, M.D., Ph.D., will discuss ongoing preclinical studies and clinical trial progress in the session “In the pipeline: updates from pharma on ongoing preclinical studies and clinical trials.”
2. Phase 1b Trial Extension and Key Results
The Phase 1b clinical trial of GT-02287 enrolled Parkinson’s patients across seven Australian sites to assess safety, tolerability and CNS exposure over three months. Initial results demonstrated central nervous system target engagement, reduction of glucosylsphingosine levels to baseline and improvement or stabilization of MDS-UPDRS scores, with an extension allowing up to 12 months of treatment.
3. Preclinical Efficacy in Parkinson’s Models
In rodent models of both GBA1-associated and idiopathic Parkinson’s disease, GT-02287 restored glucocerebrosidase function, reduced ER stress, lysosomal and mitochondrial pathology, and prevented motor and behavioral deficits including gait impairments and nesting activity decline.
4. Pipeline Expansion and Funding Support
Gain’s Magellan™ platform supports discovery of allosteric small molecule modulators for neurodegenerative and rare genetic disorders. GT-02287 has also received funding from The Michael J. Fox Foundation, The Silverstein Foundation and the Eurostars-2 program, and is being evaluated for additional indications including Gaucher’s disease and dementia with Lewy bodies.