What Endogena does

Endogena Therapeutics is a clinical-stage biotech company developing first-in-class endogenous regenerative medicines — small molecules that selectively activate endogenous adult stem and progenitor cells to drive controlled tissue repair. Founded in August 2016 by Matthias Steger and Gisbert Schneider, the company is headquartered in Zürich, Switzerland, with a registered office in San Francisco and an R&D facility at JLABS in Toronto. Its core platform combines AI-driven drug discovery with phenotypic and functional screening to identify compounds targeting molecular pathways that regulate retinal stem cells and retinal pigment epithelial (RPE) cells, with a gene-independent treatment approach that distinguishes it from mutation-specific gene therapies.

The lead clinical asset, EA-2353, is a first-in-class small molecule for retinitis pigmentosa currently in a Phase 1/2a trial (NCT05392751) that has completed dose escalation and enrollment of 14 patients across five US sites, with FDA Orphan Drug Designation (May 2021) and Fast Track Designation (February 2023). A second asset, EA-2351, received FDA IND clearance in October 2023 for geographic atrophy, an advanced form of age-related macular degeneration, with first-in-human studies planned for 2024. An earlier-stage idiopathic pulmonary fibrosis program also carries FDA Orphan Drug and Fast Track designations. The company is backed by anchor investors Centenara Labs AG and DEFTA Partners, having raised approximately $29 million across 2021. Etienne Binant serves as Interim CEO following the founder transition.

Endogena has no commercial products and generates no product revenue; its business model is R&D-driven with future value dependent on clinical proof-of-concept, regulatory approval, and eventual commercialization or out-licensing of its small-molecule regenerative assets. Strategic alliances with the Centre for Commercialization of Regenerative Medicine (CCRM), the University of Toronto, and clinical CRO InFocus Clinical Research support its translational and clinical operations, while revenue mechanics remain pre-commercial with no disclosed pricing or partnership economics.

Name

Endogena

Legal name

Endogena Therapeutics Inc.

Website

https://endogena.com

Company type

Private

Founded year

2016

Operating status

Operating

Headcount range

1–10 employees

Short description

Endogena Therapeutics is a clinical-stage biotech developing first-in-class small-molecule regenerative medicines that selectively activate endogenous adult stem and progenitor cells, with lead programs in retinitis pigmentosa (Phase 1/2a) and geographic atrophy (IND cleared).

Ownership category

akta.pro rank

Product category

Regenerative Medicine Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Transplantation & Advanced Therapies Pharmaceuticals (incl. Cell & Gene Therapies) (HLAIAAAO)

akta.pro secondary industry

Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL)

Keywords

• Regenerative medicine
• Small molecule drugs
• Ophthalmic therapeutics
• Stem cell activation
• AI drug discovery

Headquarters

HQ city

San Francisco

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Distribution channels1 record

Marketing channels6 records

Core offering

Endogena Therapeutics is a clinical-stage biotech company that discovers and develops first-in-class small-molecule drug candidates designed to selectively activate endogenous adult stem and progenitor cells for controlled tissue repair. Its lead clinical programs are EA-2353 for retinitis pigmentosa (Phase 1/2a, NCT05392751) and EA-2351 for geographic atrophy secondary to age-related macular degeneration (FDA IND cleared October 2023). The drug candidates are discovered using an AI-driven phenotypic and functional screening platform.

Product overview

Endogena Therapeutics is a clinical-stage biotech company developing first-in-class endogenous regenerative medicines. The company's core platform leverages artificial intelligence-driven drug discovery combined with phenotypic screening to identify small molecules that selectively activate endogenous stem and progenitor cells. The lead product EA-2353 is in Phase 1/2a clinical trials for retinitis pigmentosa (with FDA Orphan Drug and Fast Track designations), while EA-2351 received FDA IND clearance for geographic atrophy (AMD) with first-in-human studies planned for 2024. Earlier programs target idiopathic pulmonary fibrosis and hematopoietic recovery.

Differentiator

Problem solved

Functional benefit

Products and services

• EA-2353 First-in-class small molecule drug candidate that selectively activates endogenous retinal stem and progenitor cells to preserve and restore visual function in patients with retinitis pigmentosa. Granted FDA Orphan Drug Designation (May 2021) and Fast Track Designation (February 2023); currently in a Phase 1/2a trial that has completed enrollment of 14 patients across five US sites with no clinically relevant or dose-limiting adverse events observed.
• EA-2351 Novel small-molecule drug candidate targeting retinal pigment epithelial (RPE) cells for the treatment of geographic atrophy, an advanced form of age-related macular degeneration. FDA IND cleared in October 2023 with first-in-human study planned for 2024.
• Endogenous Regenerative Medicine Platform Proprietary drug discovery platform that uses phenotypic and functional screening combined with artificial intelligence to identify small molecules that selectively regulate endogenous adult stem and progenitor cells for controlled tissue repair across multiple degenerative disease indications.

Quantifiable outcome

• Phase 1/2a trial completed dose escalation with no clinically relevant or dose-limiting adverse events identified after repeated intravitreal injections
• +3 more outcomes

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability1 record

Feature4 records

Partnerships

Four partnerships are on record, tiered core and supporting.

• Centre for Commercialization of Regenerative Medicine (CCRM)coreStrategic or Co-development PartnerNot-for-profit, public-private consortium supporting development of foundational technologies that accelerate commercialization of cell and gene therapies and regenerative medicine technologies. Endogena is listed as an investor/partner, benefiting from Canadian scientific excellence in stem cells and bioengineering.
• University of TorontocoreStrategic or Co-development PartnerResearch collaboration with University of Toronto partners including Prof. Derek van der Kooy. Jason Charish, Director of Translational Medicine, received MSc and PhD from University of Toronto and did post-doctoral work at University Health Network. Collaboration on small-molecule-directed endogenous regeneration study published in International Journal of Molecular Sciences.
• InFocus Clinical ResearchsupportingStrategic or Co-development PartnerClinical research organization collaborating with Endogena for execution of Phase 1/2a trial of EA-2353 for retinitis pigmentosa. Credited for outstanding execution enabling trial enrollment completion ahead of schedule.
• JLABS TorontosupportingOthersEndogena maintains a research facility at JLABS in Toronto, Canada. JLABS provides lab space and resources for early-stage biotech companies.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Iveric Bio (Astellas): Developer of avacincaptad pegol (Izervay) for geographic atrophy, now part of Astellas. Direct GA competitor to Endogena's EA-2351 program and a potential strategic acquirer of complementary retinal assets.
• ProQR Therapeutics: Clinical-stage RNA editing biotech developing therapies for inherited retinal diseases including retinitis pigmentosa and Leber congenital amaurosis. Direct peer to Endogena in targeting RP via a genetic/molecular approach.
• Editas Medicine: Clinical-stage CRISPR gene editing company with ophthalmology programs including EDIT-101 for Leber congenital amaurophy and broader retinal disease pipeline. Competes for the same patient population and BD attention as Endogena.
• Gensight Biologics: Clinical-stage gene therapy company developing GS030 for retinitis pigmentosa via optogenetics. Competes directly with Endogena in the RP treatment space with a different (gene therapy) modality.
• Apellis Pharmaceuticals: Commercial-stage biotech with pegcetacoplan approved for geographic atrophy (GA). Competes directly with Endogena's EA-2351 in the same GA indication via an anti-complement mechanism.
• Nanoscope Therapeutics: Clinical-stage gene therapy company developing optogenetic therapies (MCO-010) for retinitis pigmentosa. Direct RP competitor to Endogena with a different (gene therapy/optogenetic) modality.

Emerging players

• SparingVision: Clinical-stage biotech developing SPVN06 for retinitis pigmentosa using a gene-independent AAV approach. Closely comparable to Endogena's gene-independent EA-2353 strategy but in the gene therapy modality.
• 4D Molecular Therapeutics: Clinical-stage gene therapy company with ophthalmology programs including 4D-150 for wet AMD and other retinal indications. Comparable as a retinal-disease-focused clinical biotech competing for BD capital.
• Alkeus Pharmaceuticals: Clinical-stage biotech developing oral gildeuretinol for Stargardt disease and other retinal degenerative conditions. Comparable as a small-molecule approach to inherited retinal degeneration — the same therapeutic strategy Endogena pursues.

Broad incumbents

• Regeneron Pharmaceuticals: Major ophthalmology player with Eylea (aflibercept) franchise in retinal disease including wet AMD. Sets the commercial and pricing benchmark for advanced retinal therapies and represents a likely strategic acquirer of late-stage assets like Endogena's.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks7 records

Key highlights7 records

Customer concentration

• YouTubeyoutube.com/channel/UCKj8rgJOTmNoDlWZp2qs2dA
• LinkedInlinkedin.com/company/endogena

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds2 records

Investors2 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

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

Frequently asked questions about Endogena