Gain Therapeutics, Inc. (GANX)

NASDAQ · Pharmaceuticals & Biotech · Pharmaceutical Preparations

Price
$1.95
Day
+5.98%
Market cap
$85.11M

Close 2026-09-22.

The company

Overview We are a biotechnology company developing novel small molecule therapeutics to treat diseases across several therapeutic areas, including central nervous system (“CNS”) disorders, lysosomal storage disorders (“LSDs”), metabolic disorders, and other diseases that can be targeted through protein degradation, such as oncology. GT-02287 - our lead product candidate, for the treatment of Parkinson’s disease, is currently being evaluated in a Phase 1b study in people with Parkinson’s disease with or without a GBA1 mutation. We have generated an extensive preclinical data package providing evidence of the mechanism of action and safety of GT-02287. In preclinical models of GBA1 and idiopathic Parkinson’s disease, GT-02287 has been shown to restore glucocerebrosidase, or GCase, function in the lysosome, improve mitochondrial health, reduce toxic lipid substrates and toxic forms of alpha-synuclein, reduce neuroinflammation, improve survival of dopaminergic neurons, increase dopamine levels, completely restore locomotor function and improve cognition, and reduce plasma-based neurodegeneration maker, neurofilament light chain (NfL) back to the level of healthy animals. In September 2024, we reported results from a first-in-human Phase 1 clinical trial completed in Australia to assess the safety, tolerability, pharmacokinetics, and food effect of GT-02287 in healthy participants.

From Item 1: Overview.

Institutional holders

Largest positions reported on Form 13F for the quarter ended 2026-06-30, by value.

Insider trades

Open-market buys and sales filed on Form 4, newest first. Grants, option exercises and tax withholding are left out.

Recent filings