Gain Therapeutics is developing pharmacological chaperones using AI-supported 3D structural biology and proprietary physics-based models to target allosteric binding sites on proteins implicated in rare metabolic disorders. Their lead drug candidate, GT-02287, aims to treat GBA-Parkinson’s disease and other neurodegenerative conditions by modulating protein function to restore cellular homeostasis.
Funding
$10.1M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many rare metabolic and neurodegenerative disorders are caused by misfolded or dysfunctional proteins. Traditional drug discovery methods often fail to identify effective treatments because they struggle to target the allosteric binding sites that can modulate protein function. This leaves a significant unmet need for therapies that can restore cellular homeostasis in these conditions.
Solution
Gain Therapeutics is developing a new class of allosteric small molecule therapeutics for disorders caused by dysfunctional proteins, leveraging its Magellan™ drug discovery platform. The platform combines AI-supported 3D structural biology with proprietary physics-based models to identify novel allosteric binding sites on disease-implicated proteins. These small molecules can modulate protein function through stabilization, destabilization, or degradation, based on disease pathology. Their lead drug candidate, GT-02287, is currently in Phase I clinical trials for the treatment of GBA-Parkinson’s disease and other neurodegenerative diseases.
Target Audience
The primary target audience includes patients suffering from rare metabolic disorders and neurodegenerative diseases, as well as pharmaceutical companies seeking novel drug candidates for these conditions.
Features
- Magellan™ platform uses AI-supported 3D structural biology to identify novel allosteric binding sites.
- Proprietary physics-based models pinpoint druggable pockets that are missed by current technologies.
- Allosteric small molecules can modulate protein function through various mechanisms, including stabilization and degradation.
- Lead drug candidate GT-02287 is in Phase I clinical trials.