What Gain Therapeutics does

Gain Therapeutics is a clinical-stage biotechnology company developing allosteric small molecule therapies for Parkinson's disease and other neurodegenerative and rare lysosomal storage disorders. The company was founded in 2017 in Lugano, Switzerland as Gain Therapeutics SA, with a US parent entity (Gain Therapeutics, Inc., incorporated in Maryland) established in 2020 alongside a $10 million Series B financing that brought the Swiss entity in as a wholly owned subsidiary. The company is publicly traded on NASDAQ under ticker GANX, headquartered in Bethesda, Maryland, and maintains operational offices in Lugano and Barcelona, with clinical operations in Australia and planned US expansion.

The company's core technology is the Magellan™ drug discovery platform, which combines AI-supported 3D structural biology with supercomputer-powered proprietary physics-based models to identify novel allosteric binding sites on disease-implicated proteins. These structurally targeted allosteric regulators (STARs) can modulate proteins through stabilization, destabilization, degradation, inhibition, or activation depending on disease pathology. The lead drug candidate, GT-02287, is an orally administered, brain-penetrant small molecule allosteric modulator of glucocerebrosidase (GCase) currently in Phase 1b clinical trials for Parkinson's disease with or without GBA1 mutation. A second pipeline asset, GT-04686, is a next-generation GCase allosteric modulator positioned for IND-enabling studies. Phase 1b interim data in 2026 demonstrated an 81% reduction in glucosylsphingosine in participants with elevated baseline and stable MDS-UPDRS scores over 150 days of dosing.

Gain Therapeutics operates a pre-revenue business model with no commercialized products and no disclosed pricing arrangements. The company has been funded through a €2.5 million Series A (2019), $10 million Series B (2020), a $10.1 million combined public offering and private placement (November 2023), and a $7.0 million public offering (July 2025), supplemented by approximately CHF 4.75 million in non-dilutive grants from Innosuisse and Eurostars-2/EU Horizon 2020. Grants and partnerships with the Michael J. Fox Foundation, The Silverstein Foundation for Parkinson's with GBA, University of Maryland School of Medicine, Institute for Research in Biomedicine (IRB Barcelona), Neuro-Sys SAS, and Sumitomo Dainippon Pharma provide external scientific validation and research collaboration. Go-to-market activity is focused on scientific conference presentations (AD/PD, GBA1 Meeting), investor conferences (ROTH, Oppenheimer, H.C. Wainwright), and KOL engagement, with FDA IND clearance targeted for Q2 2026 to enable US Phase 2 sites.

Name

Gain Therapeutics

Legal name

Gain Therapeutics, Inc.

Website

https://gaintherapeutics.com

Company type

Public

Founded year

2017

Operating status

Operating

Headcount range

11–50 employees

Short description

Gain Therapeutics is a clinical-stage biotech developing allosteric small molecule therapies led by GT-02287 for Parkinson's disease, using its AI-supported Magellan™ platform to identify novel binding sites on disease-implicated proteins.

Ownership category

akta.pro rank

Product category

Biopharmaceuticals — Neurodegenerative Disease Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Drug Discovery (Target ID/Validation & Hit Discovery) (HLAIALAA)

akta.pro secondary industries

Cheminformatics & Molecular Modeling / CADD (HLAGAJAE), Lead Optimization & Preclinical Candidate Selection (HLAIALAB), Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI)

Keywords

• Allosteric drug discovery
• Small molecule therapeutics
• Parkinson's disease treatment
• Lysosomal storage disorders
• AI drug discovery platform

Headquarters

HQ city

Bethesda

HQ country

United States

HQ region

North America

Offices4 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Others

Revenue model

1. Pharmaceutical Development: Pre-revenue clinical-stage biotechnology company developing allosteric small molecule therapies. The company has not yet commercialized any products and is focused on advancing GT-02287 through clinical development for Parkinson's disease.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

Gain Therapeutics is a clinical-stage biotechnology company developing orally administered, brain-penetrant allosteric small molecule therapeutics for Parkinson's disease and other neurodegenerative conditions. Its lead candidate GT-02287 is a GCase enzyme modulator currently in Phase 1b clinical trials, with the broader pipeline discovered using the proprietary Magellan™ platform that combines AI-supported 3D structural biology with supercomputer-powered physics-based modeling to identify novel allosteric binding sites on disease-implicated proteins.

Product overview

Gain Therapeutics is a clinical-stage biotechnology company developing allosteric small molecule therapies. The company's lead product is GT-02287, an orally administered brain-penetrant small molecule currently in Phase 1b clinical trials for Parkinson's disease with or without GBA1 mutation. The company is also advancing GT-04686, a next-generation GCase allosteric modulator ready for IND-enabling studies. Both programs were discovered using Gain's Magellan™ proprietary drug discovery platform, which combines AI-supported 3D structural biology with physics-based modeling to identify novel allosteric binding sites.

Differentiator

Problem solved

Functional benefit

Brands

• Magellan™: Gain's proprietary drug discovery platform that leverages AI-supported 3D structural biology and supercomputer-powered proprietary physics-based models to identify novel allosteric binding sites on disease-implicated proteins.

Products and services

• GT-02287 GT-02287 is an orally administered, brain-penetrant small molecule allosteric enzyme modulator that restores the function of glucocerebrosidase (GCase) for the treatment of Parkinson's disease with or without GBA1 mutation. Currently in Phase 1b clinical trials with interim data showing 81% reduction in glucosylsphingosine in patients with elevated baseline.
• GT-04686 GT-04686 is a next-generation glucocerebrosidase (GCase) allosteric modulator identified using the Magellan platform, positioned for IND-enabling studies for treatment of Parkinson's disease and other neurological disorders.

Quantifiable outcome

• 81% reduction in glucosylsphingosine (GluSph) in participants with elevated baseline levels after 90 days of treatment
• +3 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Five partnerships are on record, tiered core and minor.

• Institute for Research in Biomedicine (IRB)coreStrategic or Co-development Partner · 15 December 20203-year research collaboration funded by Innosuisse (CHF 850,000 of CHF 1.5M project) to further develop SEE-Tx technology for treatment of rare genetic and neurodegenerative diseases. Dr. Maurizio Molinari leads protein quality control research.
• University of Maryland School of MedicinecoreStrategic or Co-development Partner · 30 November 2020Research collaboration to investigate Gain's STAR candidates in cellular models of neuronopathic Gaucher disease and Parkinson's disease. Research led by Dr. Ricardo A. Feldman using iPSC models to study mechanism of action and therapeutic efficacy.
• Sumitomo Dainippon Pharma Co., LtdminorStrategic or Co-development Partner · 9 September 2020Research collaboration to advance Gain's Lysosomal Enzyme Allosteric Regulator Program, leveraging Sumitomo's expertise in drug development and commercialization.
• Neuro-Sys SASminorStrategic or Co-development PartnerResearch collaborator in Eurostars-2 consortium for developing innovative cures for orphan indications using preclinical in vitro models of neurodegenerative diseases.
• Clinical Advisory Board MemberscoreOthersComprised of leading Parkinson's disease experts including Karl Kieburtz (University of Rochester), Roy Alcalay (Tel Aviv Sourasky Medical Center), Samuel Broder (former NCI Director), and Wilma Van de Berg (Amsterdam UMC). Provides guidance on trial design, genetics, and biomarkers.

Scale indicators12 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Denali Therapeutics: Clinical-stage biotech developing therapies for neurodegenerative diseases, including programs targeting GBA1-Parkinson's (e.g., DNL151/tavapadon pathway). Directly comparable in indication focus, mechanism (targeting lysosomal/GCase biology), and clinical stage.
• Prevail Therapeutics (Eli Lilly subsidiary): Acquired by Eli Lilly for ~$880M in 2021 to advance AAV9-based gene therapy for GBA1-Parkinson's. Direct competitor in GBA1-PD with substantially greater resources post-acquisition.

Emerging players

• Vanqua Bio: Private biotech developing small molecule activators of GCase for Parkinson's disease and related synucleinopathies. Closely comparable mechanism (GCase activation) to GT-02287, though at an earlier stage.

Broad incumbents

• Prothena Corporation: Clinical-stage biotech with a portfolio of protein immunotherapies for neurodegenerative diseases including Parkinson's. Competes for capital, talent, and partner attention in the broader PD therapeutic space.
• Schrödinger, Inc. Computational drug discovery company whose physics-based platform overlaps with Gain's Magellan™ approach. While broader in scope (multiple therapeutic areas), it represents the closest publicly traded AI/structural-biology peer in drug discovery.
• Recursion Pharmaceuticals: AI-driven drug discovery company combining high-throughput biology with machine learning. Comparable in the AI-enabled drug discovery theme, though broader in therapeutic area coverage and target identification approach.
• BioMarin Pharmaceutical: Established rare disease biotech with approved enzyme replacement therapies for lysosomal storage disorders (e.g., Gaucher's, Pompe). Overlaps with Gain's LSD focus and provides a comparable commercial-stage benchmark.
• Ultragenyx Pharmaceutical: Clinical and commercial-stage rare disease biotech developing therapies for genetic and metabolic disorders including lysosomal storage diseases. Comparable in LSD therapeutic area, though broader in modality and stage.
• BridgeBio Pharma: Commercial-stage biotech with a portfolio of rare disease programs including CNS and metabolic disorders. Comparable in targeting rare genetic diseases with high unmet need and pursuing innovative small molecule approaches.

Regional players

• SAGE Therapeutics: CNS-focused biotech developing therapies for neurological disorders. Comparable in CNS therapeutic focus and small-molecule allosteric modulation approach (e.g., GABA-A allosteric modulators), though targeting different indications.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat3 records

Key risks6 records

Key highlights6 records

Customer concentration

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• LinkedInlinkedin.com/company/gain-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Subsidiaries1 record

Funding overview

Funding rounds6 records

Investors6 records

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M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Gain Therapeutics