BlueRock Therapeutics

What BlueRock Therapeutics does

BlueRock Therapeutics is a clinical-stage regenerative medicine company developing engineered cell therapies to replace cells lost or damaged in degenerative diseases. The company operates a Cell + Gene Platform built on pluripotent stem cell (PSC) technology, combining induced pluripotent stem cell (iPSC) reprogramming with CRISPR-based gene editing to engineer cell types with enhanced therapeutic stability and engraftment. Its lead clinical program, bemdaneprocel (BRT-DA01), consists of human embryonic pluripotent stem cell-derived dopaminergic neuron precursors surgically implanted into the striatum, with the aim of restoring motor and non-motor function in Parkinson's disease; this candidate is currently in a registrational Phase III trial (exPDite-2) following positive 36-month Phase I data showing a 17.9-point mean MDS-UPDRS Part III reduction in the high-dose cohort. The second clinical program, OpCT-001, is an iPSC-derived photoreceptor cell therapy for inherited retinal disorders such as retinitis pigmentosa, currently in a Phase 1/2a trial (CLARICO). Earlier-stage work also targets retinal pigment epithelium (RPE) cell therapy for dry age-related macular degeneration.

The company was founded in 2016 as a joint venture between Versant Ventures and Leaps by Bayer with a $225M Series A, and was acquired in full by Bayer AG in late 2019, becoming a wholly owned, independently managed subsidiary forming the cornerstone of Bayer's Cell & Gene Therapy Platform. Scientific co-founders include Dr. Lorenz Studer (Memorial Sloan Kettering Cancer Center) and Dr. Gordon Keller (McEwen Centre for Regenerative Medicine at University Health Network in Toronto), anchoring the platform's stem cell biology expertise. The company is headquartered in Cambridge, Massachusetts, with R&D and manufacturing operations in Toronto (including a long-term ~80,000 sq ft anchor lease at the Catalyst life sciences facility), clinical and scientific collaboration in New York (Memorial Sloan Kettering), and a German entity in Berlin. Strategic technology and clinical-manufacturing collaborations include Opsis Therapeutics / FUJIFILM Cellular Dynamics on OpCT-001 manufacturing, Editas Medicine on CRISPR gene editing, and Senti Biosciences on gene-circuit engineering.

BlueRock does not currently generate product revenue and operates as a clinical-stage, pre-commercial biotechnology company. Future revenue, contingent on regulatory approval, would derive from commercial sales of approved cell therapies administered in specialty hospital and treatment-center settings under a traditional specialty pharma commercialization model, with a one-time-per-patient dosing paradigm rather than recurring dosing. Capital and operational burn are funded by its parent Bayer AG, and standalone financial disclosures are not produced because BlueRock's results are consolidated within Bayer's reporting. Three pipeline assets carry FDA designations (Fast Track, RMAT for bemdaneprocel; Fast Track and Orphan Drug for OpCT-001), and bemdaneprocel additionally holds Japan's SAKIGAKE Pioneering Regenerative Medical Product designation, signalling expedited pathways in both major regulatory jurisdictions.

Name

BlueRock Therapeutics

Legal name

BlueRock Therapeutics LP

Website

https://bluerocktx.com

Company type

Private

Founded year

2016

Operating status

Operating

Headcount range

251–500 employees

Short description

BlueRock Therapeutics is a clinical-stage regenerative medicine company developing engineered cell therapies for degenerative diseases, with a Phase III Parkinson's candidate (bemdaneprocel) and a Phase 1/2a photoreceptor disorder program (OpCT-001), operating as a wholly owned subsidiary of Bayer AG.

Ownership category

akta.pro rank

Product category

Cell Therapy / Regenerative Medicine

NAICS

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

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

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

akta.pro secondary industries

Stem Cell–Based Regenerative Therapies (HLAAAGAA), Cell Therapy Developers (Allogeneic/Off-the-shelf) (HLAAABAB), Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL)

Keywords

• Cell therapy development
• Regenerative medicine
• Pluripotent stem cell therapy
• Neurological disease therapy
• Ophthalmology cell therapy

Headquarters

HQ city

Toronto

HQ country

Canada

HQ region

North America

Offices5 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Cell Therapy Development and Commercialization: As a clinical-stage biopharmaceutical company, BlueRock Therapeutics does not currently generate revenue. The company is developing cell therapy products for neurological and ophthalmological diseases. If regulatory approvals are obtained, future revenue would come from commercial sales of cell therapy products such as bemdaneprocel for Parkinson's disease and OpCT-001 for primary photoreceptor diseases.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels9 records

Core offering

BlueRock Therapeutics develops investigational allogeneic cell therapies derived from pluripotent stem cells (iPSCs and hESCs) for degenerative diseases. Its clinical pipeline includes bemdaneprocel, a dopaminergic neuron replacement therapy in pivotal Phase III for Parkinson's disease, and OpCT-001, an iPSC-derived photoreceptor cell therapy in Phase 1/2a for inherited retinal diseases. Therapies are built on a proprietary Cell+Gene Platform combining stem cell biology with gene editing to enhance therapeutic effect and stability.

Product overview

BlueRock Therapeutics is a clinical-stage cell therapy company developing cellular medicines for degenerative diseases. The company operates a Cell + Gene Platform that utilizes pluripotent stem cells (PSCs) - primarily induced pluripotent stem cells (iPSCs) and human embryonic stem cells - to create highly specific cell types for replacement therapies. The clinical pipeline includes bemdaneprocel (BRT-DA01), a Phase III cell therapy for Parkinson's disease targeting dopaminergic neuron replacement, and OpCT-001, a Phase 1/2a iPSC-derived therapy for primary photoreceptor diseases. Programs focus on neurology (Parkinson's disease) and ophthalmology (inherited retinal disorders).

Differentiator

Problem solved

Functional benefit

Products and services

• bemdaneprocel (BRT-DA01) Investigational allogeneic cell therapy derived from human embryonic pluripotent stem cells. Dopaminergic neuron precursors are surgically implanted into the brain to replace dopamine-producing neurons lost in Parkinson's disease, with the goal of reforming neural networks and restoring motor and non-motor function. Currently in Phase III clinical development with FDA Fast Track (2021) and Regenerative Medicine Advanced Therapy (RMAT) designations and SAKIGAKE designation in Japan.
• OpCT-001 Investigational induced pluripotent stem cell (iPSC)-derived cell therapy for primary photoreceptor diseases including retinitis pigmentosa, Usher syndrome, and cone-rod dystrophy. Aims to restore vision by replacing degenerated photoreceptor cells in the retina. Currently in Phase 1/2a (CLARICO) with FDA Fast Track and Orphan Drug designations. Developed in collaboration with Opsis Therapeutics and FUJIFILM Cellular Dynamics.

Quantifiable outcome

• Phase I 36-month data: High dose cohort showed mean reduction of 17.9 points on MDS-UPDRS Part III (OFF-medication state) compared to baseline; mean increase of 1 hour in 'Good ON' state time; mean decrease of 0.93 hours in 'OFF' state time.
• +2 more outcomes

Named customers2 records

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Seven partnerships are on record, tiered strategic and core.

• Michael J. Fox Foundation for Parkinson's ResearchstrategicGTM or Marketing Partner · 1 January 2026Premier event sponsor of Michael J. Fox Foundation's 'Parkinson's IQ + You' national educational event series. The events educate and empower people living with Parkinson's disease and their families through interactive panels and research discussions.
• Foundation Fighting BlindnessstrategicGTM or Marketing Partner · 1 January 2026Gold sponsor of Prevent Blindness's Inherited Retinal Disease and Genetic Testing Month. Sponsored producer of Foundation Fighting Blindness's Eye on the Cure Podcast. Supports patient education and awareness for inherited retinal diseases.
• Opsis Therapeutics / FUJIFILM Cellular DynamicscoreStrategic or Co-development Partner · 1 January 2024Exclusive licensing and collaboration agreement with Opsis Therapeutics LLC and FUJIFILM Cellular Dynamics, Inc. (FCDI) for OpCT-001, an iPSC-derived cell therapy for primary photoreceptor diseases. The collaboration includes research, development, and clinical manufacturing of OpCT-001. FCDI supported IND-enabling activities and clinical manufacturing. Opsis Therapeutics is a joint venture focused on cell replacement therapies for retinal diseases.
• Editas MedicinestrategicTechnology or Integration · 1 January 2021Collaboration leveraging Editas Medicine's CRISPR genome editing technology to develop engineered iPSC cells with enhanced therapeutic functions. The partnership aims to enable precise gene editing capabilities within BlueRock's cell therapy platform to improve therapeutic potential and stability.
• Senti BiosciencesstrategicTechnology or Integration · 1 January 2021Collaboration with Senti Biosciences, a gene circuit company, to enhance BlueRock's cell+gene platform. Senti's technology enables precise sensing and responding to disease state and therapeutic payload expression within engineered cells.
• Memorial Sloan Kettering Cancer CentercoreStrategic or Co-development Partner · 1 January 2016Scientific co-founder Dr. Lorenz Studer serves as Director of Center for Stem Cell Biology at Memorial Sloan Kettering Cancer Center. Dr. Viviane Tabar serves as Chair of Department of Neurosurgery. The institution contributes scientific expertise and research capabilities to BlueRock's cell therapy programs.
• University Health Network / McEwen Centre for Regenerative MedicinecoreStrategic or Co-development Partner · 1 January 2016Scientific co-founders Dr. Gordon Keller (Director of McEwen Centre) and Dr. Michael Laflamme (Senior Scientist at Toronto General Hospital Research Institute) are affiliated with UHN. The Toronto location houses BlueRock's research operations and manufacturing facility.

Scale indicators7 records

Recent moves8 records

Expansion highlights6 records

Others

• Editas Medicine: Editas Medicine is a gene editing partner of BlueRock (CRISPR collaboration) and a fellow clinical-stage cell/gene therapy player, making it a relevant adjacent peer and ecosystem participant.
• Senti Biosciences: Senti Biosciences is a gene circuit technology partner of BlueRock's cell+gene platform and operates in the same engineered-cell-therapy ecosystem, making it a relevant technology/partner peer.

Direct peers

• Fate Therapeutics: Fate Therapeutics is a leading iPSC-derived cell therapy company focused on off-the-shelf NK and T-cell cancer immunotherapies, employing a similar pluripotent stem cell platform and allogeneic business model to BlueRock.
• Aspen Neuroscience: Aspen Neuroscience is developing iPSC-derived autologous cell therapies for Parkinson's disease, directly competing with BlueRock's bemdaneprocel in the same indication using a closely related pluripotent stem cell platform.
• Century Therapeutics: Century Therapeutics develops allogeneic iPSC-derived immune cell therapies (NK and T cells) for cancer, sharing BlueRock's allogeneic iPSC platform approach and clinical-stage biotech profile.
• Lineage Cell Therapeutics: Lineage Cell Therapeutics develops allogeneic cell transplant therapies for neurological and ophthalmic conditions, including photoreceptor and RPE cell programs that closely mirror BlueRock's OpCT-001 strategy.

Emerging players

• Sangamo Therapeutics: Sangamo Therapeutics develops genomic medicines including cell therapy approaches using zinc finger and other genome-editing platforms, with a comparable clinical-stage profile and focus on CNS/neurological indications overlapping with BlueRock's PD program.
• CRISPR Therapeutics: CRISPR Therapeutics develops CRISPR/Cas9-based gene-edited cell therapies (including for regenerative medicine indications) and shares a board director with BlueRock via Bayer's R&D leadership, making it a closely connected emerging cell/gene peer.
• International Stem Cell Corporation (ISCO) / ViaCyte: ViaCyte develops stem cell-derived cell replacement therapies (notably for diabetes) and is part of the broader regenerative medicine ecosystem; it is comparable to BlueRock as a clinical-stage pluripotent stem cell therapy developer.

Broad incumbents

• Vertex Pharmaceuticals: Vertex has built a major cell therapy franchise including a stem cell-derived islet cell therapy for type 1 diabetes, and is a large-cap incumbent pursuing regenerative cell therapy approaches that overlap with BlueRock's PSC-based strategy.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/bluerocktx
• Xx.com/BlueRockTx
• Facebookfacebook.com/417392058783123

Compliance4 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles18 records

Funding overview

Funding rounds1 record

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 BlueRock Therapeutics