Opsis Therapeutics

What Opsis Therapeutics does

Opsis Therapeutics is a private clinical-stage biotechnology company founded in 2016 as a joint venture between FUJIFILM Cellular Dynamics and Dr. David Gamm, M.D., Ph.D., headquartered in Madison, Wisconsin. The company develops iPSC-derived cell replacement therapies for ocular diseases, with a pipeline targeting dry age-related macular degeneration (AMD) and primary photoreceptor diseases such as retinitis pigmentosa. Its core technology platform integrates induced pluripotent stem cell (iPSC) generation, three-dimensional optic vesicle-based retinal cell differentiation (first described in the Gamm laboratory), and functional biomaterials to produce photoreceptors and other retinal cells for transplantation. The lead candidate, OpCT-001, is an iPSC-derived cell therapy for primary photoreceptor diseases that received FDA IND clearance in September 2024, FDA Fast Track Designation in February 2025, and began dosing in the CLARICO Phase 1/2a clinical trial in July 2025.

Opsis operates a partnership- and licensing-driven business model rather than a direct commercialization model. In May 2021, the company entered a strategic R&D alliance with BlueRock Therapeutics (a Bayer subsidiary) and FUJIFILM Cellular Dynamics, which included a $70 million up-front payment from Bayer. In January 2024, BlueRock exercised its exclusive option to license OpCT-001, advancing it through clinical development and bearing downstream commercialization responsibility. Opsis therefore generates revenue through upfront payments, development and regulatory milestones, and royalties on licensed candidates, while day-to-day clinical execution is conducted by its larger partner. The company is small (1-10 employees) and is structured as Opsis Therapeutics, LLC under Wisconsin jurisdiction, with a co-founder-led operating team and a Scientific Advisory Board.

The company's strategic posture is best characterized as a focused, asset-light development organization that monetizes its platform and lead candidate via licensing to large pharma, while leveraging the iPSC capabilities of its parent (FUJIFILM Cellular Dynamics) and the academic retinal regeneration expertise of its scientific founders. With OpCT-001 now in Phase 1/2a and additional dry AMD programs in earlier-stage development, Opsis is positioned as a clinical validation-stage player in the ocular cell therapy space, with FDA Orphan Drug Designation providing potential seven years of market exclusivity upon approval.

Name

Opsis Therapeutics

Legal name

Opsis Therapeutics, LLC

Website

https://opsistx.com

Company type

Private

Founded year

2016

Operating status

Operating

Headcount range

1–10 employees

Short description

Opsis Therapeutics is a private Madison, Wisconsin-based biotech founded in 2016 as a joint venture between FUJIFILM Cellular Dynamics and David Gamm, M.D., Ph.D., developing iPSC-derived cell replacement therapies for dry AMD and primary photoreceptor diseases, with lead candidate OpCT-001 licensed to BlueRock Therapeutics and currently in Phase 1/2a clinical trials.

Ownership category

akta.pro rank

Product category

Cell Therapy for Retinal Diseases

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414), Scientific Research and Development Services (5417)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

Stem Cell Therapies (MSC/iPSC/HSC-derived) (HLAAABAE)

akta.pro secondary industries

Ophthalmic Regeneration (cornea/retina) (HLAAAGAK), Rare Ophthalmology Disorder Therapies (HLAIAIAK), Stem Cell–Based Regenerative Therapies (HLAAAGAA)

Keywords

• Cell replacement therapy
• Retinal disease treatment
• IPSC-derived therapies
• Ophthalmic biotechnology
• Photoreceptor regeneration

Headquarters

HQ city

Madison

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Supply Chain

Revenue model

1. Licensing and Strategic Partnerships: Opsis generates value through licensing its iPSC-derived cell therapy candidates to larger pharmaceutical companies. The company entered a strategic R&D alliance with BlueRock Therapeutics and FUJIFILM Cellular Dynamics, with BlueRock exercising an exclusive option to license OpCT-001. Revenue is generated through upfront payments, milestone payments, and royalties from licensed candidates.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels1 record

Core offering

Opsis Therapeutics develops iPSC-derived cell replacement therapies for ocular diseases, with a lead candidate (OpCT-001) targeting primary photoreceptor diseases and additional programs for dry age-related macular degeneration (AMD). The company's platform combines induced pluripotent stem cell (iPSC) generation, retinal cell differentiation, and functional biomaterials to create disease-modifying cell therapies administered through clinical trial sites and licensed to larger pharmaceutical partners.

Product overview

Opsis Therapeutics is developing a pipeline of cell replacement therapies targeting ocular diseases, specifically dry age-related macular degeneration (AMD) and primary photoreceptor diseases. The company's core technology platform leverages induced pluripotent stem cell (iPSC) generation, retinal cell differentiation, and functional biomaterials. Their lead candidate, OpCT-001, is an iPSC-derived cell therapy for primary photoreceptor diseases that has been licensed to BlueRock Therapeutics and is currently in Phase 1/2a clinical trials. The company was founded in 2016 as a joint venture of FUJIFILM Cellular Dynamics and David Gamm, M.D., Ph.D.

Differentiator

Problem solved

Functional benefit

Quantifiable outcome

• OpCT-001 received FDA Orphan Drug Designation, providing potential seven years of market exclusivity upon approval plus tax credits and user fee exemptions
• +1 more outcomes

Named customers1 record

Segments1 record

Ideal customer profiles1 record

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered core.

• BlueRock TherapeuticscoreStrategic or Co-development Partner · 17 May 2021BlueRock Therapeutics entered into a strategic R&D alliance with FUJIFILM Cellular Dynamics and Opsis Therapeutics to discover and develop cell therapies for eye diseases. BlueRock exercised its exclusive option in January 2024 to license OpCT-001, an iPSC-derived cell therapy for primary photoreceptor diseases. OpCT-001 is being advanced through the CLARICO Phase 1/2a clinical trial.
• FUJIFILM Cellular DynamicscoreStrategic or Co-development Partner · 1 January 2016FUJIFILM Cellular Dynamics is a co-founder of Opsis Therapeutics through a joint venture arrangement. The partnership combines FUJIFILM Cellular Dynamics' expertise in iPSC technology with Opsis Therapeutics' ocular disease focus. The joint venture leverages FUJIFILM's iPSC platform capabilities for developing cell replacement therapies.

Scale indicators3 records

Recent moves6 records

Expansion highlights4 records

Direct peers

• BlueRock Therapeutics: Bayer subsidiary that licensed OpCT-001 from Opsis Therapeutics in January 2024 and is now running the CLARICO Phase 1/2a trial. Most directly comparable as a clinical-stage cell therapy developer focused on ocular disease using iPSC-derived approaches.
• Lineage Cell Therapeutics: Clinical-stage cell therapy company developing allogeneic retinal pigment epithelium (RPE) cell transplants for dry AMD and other retinal diseases. Directly comparable as a retinal cell replacement therapy peer.
• jCyte: Clinical-stage biotech developing allogeneic human retinal progenitor cells (jCell) for retinitis pigmentosa — a direct indication overlap with OpCT-001 and a directly comparable cell therapy approach in inherited retinal disease.
• ReNeuron: UK-based stem cell therapy developer with a retinal program (hRPC cells for retinitis pigmentosa). Compares directly to Opsis in stem cell-derived photoreceptor replacement therapies for inherited retinal disease.

Others

• FUJIFILM Cellular Dynamics: Joint venture parent of Opsis Therapeutics and a leading iPSC technology platform. Operates in the same iPSC-derived cell therapy space and provides the underlying cell line and differentiation capabilities that Opsis leverages.

Broad incumbents

• Spark Therapeutics: Roche subsidiary and pioneer in gene therapy for inherited retinal diseases (e.g., LUXTURNA for RPE65-mediated IRD). Represents the dominant gene therapy incumbent competing for the same patient population as OpCT-001.
• Astellas Institute for Regenerative Medicine: Astellas' cell therapy division (formerly Universal Cells) develops stem cell-derived therapies for ophthalmology. Represents a broader incumbent with overlapping regenerative medicine capabilities in ocular indications.

Emerging players

• Editas Medicine: CRISPR gene editing company developing EDIT-101 (reniFiguren-cel) for Leber congenital amaurosis 10 — overlapping inherited retinal disease focus with Opsis, though differentiated as a gene editing rather than cell replacement approach.
• ProQR Therapeutics: RNA editing company developing sepofarsen for Leber congenital amaurosis and additional RNA therapies targeting inherited retinal diseases. Competes for the same patient population with a different modality.
• Coave Therapeutics: European gene therapy company developing AAV-based therapies for ocular diseases including retinitis pigmentosa. Targets overlapping patient population to Opsis via a gene therapy modality.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/OpsisTherapy

Revenue estimate

Valuation estimate

Number of profiles

Funding overview

Funding rounds

Investors

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 Opsis Therapeutics