COMPANY PROFILE / iPS CELLS & REGENERATIVE MEDICINE
BlueRock Therapeutics
A Bayer subsidiary transplanting cells made from embryonic and iPS cells
BlueRock Therapeutics is a wholly owned subsidiary of Bayer that develops treatments in which cells made from pluripotent stem cells are transplanted into the brain or retina. Its most advanced candidate, bemdaneprocel, is in Phase 3 for Parkinson's disease, and its starting material is embryonic stem (ES) cells. The iPS cell-derived candidate is lemiretprocel (OpCT-001), for retinal disease, which is in Phase 1/2a. As of October 2026 no product has been approved. BlueRock is unlisted and does not publish its financials, so this profile relies only on official announcements from Bayer and BlueRock, peer-reviewed papers and clinical trial registrations. It is not medical advice on treatment choices.
- BlueRock in 30 seconds, and where it stands in iPS cells and regenerative medicine
- The logo and how the name is written
- Where the company sits in the iPS cell and regenerative medicine landscape
- Scale (an unlisted subsidiary)
- Resources committed to the field
- Track record so far
- Plans ahead
- What the company is aiming for
- Partnership map
- The technologies and products it offers
- The risks this company carries
- Glossary, references and claim-to-source audit
1. BlueRock in 30 seconds, and where it stands in iPS cells and regenerative medicine
Massachusetts, U.S.238 Main Street (as stated in company releases)
Big-pharma subsidiary (secondary)Founded in 2016; Bayer took full ownership in 2019
Parkinson's disease, Phase 3ES cell-derived. No product has been approved yet
Phase 1/2aPrimary photoreceptor diseases. Originated at FUJIFILM Cellular Dynamics
R&D of €5.8 billionThe whole Bayer group in FY2025, not BlueRock alone
Companies involved in iPS cells and regenerative medicine fall into three broad groups: large pharma & strategic partners, genome editing & gene therapy, and iPS cell & cell therapy. BlueRock is an example of an "iPS cell & cell therapy" company that has become a wholly owned subsidiary of a large pharmaceutical company, Bayer. It has two clinical-stage candidates. The more advanced, bemdaneprocel, is derived from ES (embryonic stem) cells, and the other, lemiretprocel (OpCT-001), is derived from iPS cells Sourced. When Bayer announced the acquisition in 2019 it described BlueRock as built on a proprietary iPS cell platform, but the candidate that has since reached Phase 3 is, in the company's own description, ES cell-derived Sourced. So it would be inaccurate to say simply that "BlueRock is an iPS cell company". The accurate description is a company that transplants cells made from pluripotent stem cells (ES cells and iPS cells). Because it is unlisted, its revenue, R&D spending and headcount are not published. This article therefore keeps to what Bayer's disclosures and clinical trial registrations can support.
2. The logo and how the name is written
A note on names: the legal entity is BlueRock Therapeutics LP (a limited partnership). The company name does not include Bayer's name, but the company describes itself as a wholly owned subsidiary of Bayer AG that operates independently. The logo is the image used in the header of the official website, downloaded and placed here; we have not recreated it with generated imagery. The candidates are named bemdaneprocel (development code BRT-DA01, formerly MSK-DA01) and lemiretprocel (development code OpCT-001). The announcement of 30 September 2026 gave the name lemiretprocel alongside OpCT-001.
Official site: https://www.bluerocktx.com/the-science/pipeline/3. Where the company sits in the iPS cell and regenerative medicine landscape
Looked at by mechanism, BlueRock combines the following elements. The issues covered in this series' explainers on iPS cells, iPS-derived neurons and Parkinson's disease, MHC and universal donor cells, autologous versus allogeneic cells, and cell manufacturing and cell banks all appear here.
| Mechanism | How BlueRock uses it | Category |
|---|---|---|
| ES cell-derived cells | bemdaneprocel: dopamine neuron precursor cells made from human embryonic stem cells. A frozen, ready-made (off-the-shelf) product transplanted into the putamen of the brain | Sourced |
| iPS cell-derived cells | lemiretprocel (OpCT-001): photoreceptor precursor cells made from iPS cells, administered under the retina. Exclusively in-licensed in January 2024 from FUJIFILM Cellular Dynamics and Opsis | Sourced |
| Allogeneic (other-person-derived) cells | Both use cells from someone other than the patient, and need immunosuppression to hold off rejection. In the Phase 1 trial of bemdaneprocel, every participant received immunosuppression for one year | Sourced |
| Transplantation (surgery) | Transplantation into the brain is a surgical procedure. The Phase 3 trial is designed with a sham surgery as the control | Sourced |
| Gene-engineering concept | Official announcements describe both clinical candidates as "cells derived from pluripotent stem cells" and do not mention adding functions through genetic engineering. The 2019 announcement did speak of a concept of strengthening cells through genetic engineering (called CELL+GENE) | Sourced |
4. Scale (an unlisted subsidiary)
BlueRock is unlisted, and none of the primary sources we could confirm gives its revenue, R&D spending, headcount or profit and loss Not yet confirmed. We therefore show the scale of the parent, Bayer, in a single table for the group as a whole.
| Bayer group as a whole (FY2025) | Value | How the source states it |
|---|---|---|
| Sales | €45.6 billion | Boilerplate at the end of Bayer's official release |
| R&D expense | €5.8 billion | Same |
| Employees | About 88,000 | Same |
The figures above are for the Bayer group as a whole. They do not show the size of BlueRock. The only amounts we can confirm as having gone into BlueRock are the US$225 million Series A at its founding in 2016 and the terms of the 2019 acquisition (about US$240 million at closing, plus up to US$360 million more on achievement of development milestones) Sourced. We could not confirm how much of the milestone money has been paid so far Not yet confirmed.
5. Resources committed to iPS cells and regenerative medicine
We sort four points (people, money, outside investments, acquisitions and in-licensing, and links with universities and public funding) by whether the primary sources let us confirm them.
| Aspect | What the primary sources confirm | Status |
|---|---|---|
| People (sites and headcount) | Headquarters are in Cambridge, Massachusetts. BlueRock's own headcount and a list of its research sites do not appear in the company's releases | Only part of the sites confirmed. Headcount could not be confirmed |
| Money (R&D spending and manufacturing facilities) | BlueRock's own R&D spending and capital expenditure are not disclosed. In 2020 Bayer set up a cell and gene therapy platform, and it has bought BlueRock (valued at up to about US$1 billion) and AskBio (2020; US$2 billion at closing plus up to US$2 billion in milestones) | Only the acquisition amounts confirmed |
| Outside investments, acquisitions and in-licensing | Bayer made BlueRock a wholly owned subsidiary in 2019. BlueRock began working with FUJIFILM Cellular Dynamics and Opsis in 2021, and in January 2024 exclusively in-licensed OpCT-001 | Confirmed |
| Universities, research institutions and public funding | The Phase 1 trial is named MSK-DA01, but the primary sources we consulted say nothing about the terms of any agreement with a university. We also found no mention of public funding | Could not be confirmed |
The Nature paper describes bemdaneprocel as cryopreserved, ready-made precursor cells Sourced. Unlike autologous approaches, which make cells for each patient, cells can be produced in large batches in advance and frozen, which makes batch-to-batch variation easier to manage. The central quality challenge then becomes keeping viability and properties constant after freezing and thawing (this is our explanation; the company does not disclose its process). In the Phase 1 trial the low dose was 900,000 cells and the high dose 2.7 million cells, transplanted into both sides of the putamen. With so few cells, how many patients' doses one manufacturing run can yield could determine the economics, but the company does not disclose its manufacturing capacity Not yet confirmed.
For OpCT-001 (iPS cell-derived), the company writes that clinical manufacturing was carried out at the cGMP facility of FUJIFILM Cellular Dynamics in Madison, Wisconsin Sourced. In other words, the work is divided: BlueRock designs the cells and a partner makes them, so managing the contract manufacturer affects both quality and supply.
6. Track record so far
| When | What happened | What it means |
|---|---|---|
| 2016 | Versant Ventures and Bayer (Leaps by Bayer) founded BlueRock. Series A of US$225 million | A cell therapy company launched with a large pharmaceutical company as an investor |
| August 2019 | Bayer announced it would take full ownership of BlueRock. Its stake at the time was 40.8%. About US$240 million at closing plus up to US$360 million | Became a wholly owned subsidiary by the end of 2019 |
| October to December 2020 | Bayer acquired the gene therapy company AskBio and set up a cell and gene therapy platform within its Pharmaceuticals division | BlueRock is the core of the cell-therapy side |
| 2021 | bemdaneprocel received FDA Fast Track designation. Began working with FUJIFILM Cellular Dynamics and Opsis | Two pillars: Parkinson's disease and retinal disease |
| January 2024 | Exclusively in-licensed OpCT-001 from FUJIFILM Cellular Dynamics and Opsis | An iPS cell-derived candidate becomes its own |
| May 2024 | bemdaneprocel designated RMAT by the FDA | Eligible for expedited development and review |
| September 2024 | The FDA cleared the IND (application to begin clinical trials) for OpCT-001 | The company describes it as the first clinical trial of an iPS cell-derived therapy in primary photoreceptor disease |
| January 2025 | Announced plans for a Phase 3 trial of bemdaneprocel (exPDite-2) | Following the data from 12 participants in Phase 1 |
| 2025 | Phase 1 results published in Nature. exPDite-2 has a registered start date of June, and in September the first participant was treated after randomisation | Collection of the data for a marketing application begins |
| December 2025 | bemdaneprocel received SAKIGAKE designation in Japan (the Pioneering Regenerative Medical Product designation) | Allows frequent consultation with Japan's Ministry of Health, Labour and Welfare (MHLW) |
| January 2026 | OpCT-001 received FDA orphan drug designation for retinitis pigmentosa | Treated as a rare disease |
| 30 September 2026 | lemiretprocel (OpCT-001) received orphan drug designations from both the FDA and the EMA | Extended to Europe |
Note also that the company's boilerplate description gave its fields as "neurology, ophthalmology, cardiovascular and immunology" in releases up to January 2025, but in releases from September 2025 onward it lists only "neurology and ophthalmology" Sourced.
7. Plans ahead
exPDite-2 is a Phase 3 trial. On ClinicalTrials.gov its primary completion is estimated for March 2027 and completion of the whole study for March 2032 (both estimates, which may change) Sourced. The company writes that the results of this trial are expected to form part of the data package supporting marketing applications to regulators Sourced.
None of the following appears in the primary sources Not yet confirmed.
The timing of a marketing application / manufacturing capacity (how many patients can be supplied per year) / price / BlueRock's own headcount and R&D spending / the cumulative milestone payments made by Bayer / when the Phase 3 results will be released. Press-based figures and research-firm estimates are not used in this article.
8. What the company is aiming for in iPS cells and regenerative medicine
This section includes things the company does not say directly. We set them out as inferences within what the primary sources support, with the evidence for each.
| What the evidence suggests | Primary information it rests on | Category |
|---|---|---|
| It aims to restore function by replacing lost cells | The company describes bemdaneprocel as replacing the dopamine neurons that have been lost, and OpCT-001 as replacing degenerated retinal cells with functioning ones | Sourced |
| It has narrowed its focus to two areas, neurology and ophthalmology | Releases from 2024 to January 2025 list neurology, ophthalmology, cardiovascular and immunology; from September 2025 only neurology and ophthalmology | Sourced |
| Cardiovascular and immunology may have become lower priorities | The change in the boilerplate description above. No reason is given | Inference |
| Its policy is to share manufacturing with outside specialist companies | FUJIFILM Cellular Dynamics handled clinical manufacturing of OpCT-001 and is also the source from which the iPS cell-derived candidate was in-licensed | Inference |
| It intends to proceed in parallel with several regulators, including Japan's | It has obtained, one after another, Japan's SAKIGAKE designation, orphan designation from the EMA, and RMAT and Fast Track from the FDA | Inference |
When it was acquired in 2019, BlueRock put its "iPS cell platform" to the fore Sourced. Yet the candidate that has moved on to Phase 3, bemdaneprocel, is ES cell-derived, and the iPS cell-derived OpCT-001 is at the Phase 1 stage Sourced. From this we read that what matters to BlueRock is less the origin of the cells (ES or iPS) than which disease the cells are meant to replace cells in. If techniques that make it easier to hold off rejection of other-person-derived iPS cells become practical, moving from ES cells to iPS cells on the same logic could become an option. That is our own speculation and does not appear in any company announcement Inference.
9. Partnership map for iPS cells and regenerative medicine
The most important of BlueRock's relationships is the one with its parent, Bayer. It is described as independently operated, but the acquisition amounts, the creation of the platform and the announcements of designations all appear in Bayer's releases Sourced. The other is the relationship with FUJIFILM Cellular Dynamics: for the iPS cell-derived candidate, the structure is one in which the source of the licence is also the manufacturer. Read together with our FUJIFILM Cellular Dynamics profile, it shows how the same candidate looks from each company's side.
10. The technologies and products it offers
There is no approved product. The candidates in development are listed below. All are unapproved investigational products, and neither efficacy nor safety has been established.
| Candidate | Cell source and target | Stage (trial name, NCT number) | Primary endpoint |
|---|---|---|---|
| bemdaneprocel (BRT-DA01) | Dopamine neuron precursor cells derived from human ES cells. Parkinson's disease (moderate) | In development (Phase 3). exPDite-2, NCT06944522. About 102 people, randomised, sham-surgery-controlled, double-blind. FDA Fast Track (2021) and RMAT (2024); Japan's SAKIGAKE (2025) | Change at week 78 in ON time without dyskinesia (patient diary) |
| bemdaneprocel Phase 1 | As above. 12 people | Completed. MSK-DA01, NCT04802733. Open-label, single arm. Long-term follow-up is NCT05897957 | Safety and tolerability |
| lemiretprocel (OpCT-001) | iPS cell-derived photoreceptor precursor cells. Primary photoreceptor diseases such as retinitis pigmentosa and cone-rod dystrophy | In development (Phase 1/2a). CLARICO, NCT06789445. Enrolling. FDA Fast Track; orphan drug designation from the FDA and EMA | Ocular and systemic adverse events up to week 52 |
| Other research-stage candidates | A next-generation Parkinson's disease candidate (BR-DA02), microglia, oligodendrocytes, retinal pigment epithelium, and photoreceptors combined with retinal pigment epithelium | Preclinical and early development (as listed in the pipeline table on the official website) | - |
The published results all come from a small Phase 1 trial and do not guarantee approval or treatment benefit.
| Result | Figure | Comparator and time point |
|---|---|---|
| bemdaneprocel Phase 1 (safety) | Met its primary objective of safety and tolerability. No adverse events related to the cell product (Nature). The company says there were no serious adverse events related to the cell product at 24 and 36 months | 12 people. Open-label, single arm, no comparator. At 1 year (primary) and 18 months (imaging) |
| bemdaneprocel Phase 1 (motor symptoms) | Average improvement of 23 points in MDS-UPDRS Part III (off medication) in the high-dose group (Nature) | 7 in the high-dose group and 5 in the low-dose group. Being open-label, it cannot rule out effects such as placebo response. At 18 months, versus baseline |
| bemdaneprocel Phase 1 (imaging) | Increased 18F-DOPA PET uptake in the putamen (suggesting survival of the graft) | At 18 months |
In transplantation to the brain or retina, quality depends not only on the cells themselves but on the fluid in which they are suspended, the cryopreservation medium, and the needles and catheters used for injection. The Phase 1 trial required surgery to transplant cells into both sides of the putamen (Nature). The sham surgery in the Phase 3 trial can be read as a design to separate out the placebo effect that comes with the operation (our explanation). OpCT-001 is administered under the retina, and because the retina is extremely thin tissue, the volume and speed of injection and suspension conditions that prevent cell aggregation are likely to be the practical keys; the company does not disclose specific materials or devices Not yet confirmed.
11. The risks this company carries
| Risk | Details | Category |
|---|---|---|
| No approved product yet | bemdaneprocel is in Phase 3, and the outcome could change the whole outlook. The company itself states plainly that it has not been approved by any regulator | Sourced |
| Limits of the Phase 1 results | An open-label, single-arm study of 12 people; the improvement in motor symptoms was not compared with a control. The Phase 3 sham-surgery control is designed to answer that | Sourced |
| Immunosuppression is a precondition | Because the cells are allogeneic, every participant in Phase 1 received immunosuppression for one year. Whether it can be reduced is a future question | Sourced |
| Surgical risk and a narrow target population | Transplantation into the brain is surgery. Phase 3 targets moderate disease in people aged 45 to 75 | Sourced |
| One candidate depends on a partner for manufacturing | FUJIFILM Cellular Dynamics carried out clinical manufacturing of OpCT-001. Details of the supply arrangement are not disclosed | Sourced |
| An unlisted company with limited information | Revenue, headcount, R&D spending and cash position cannot be verified from outside | Not yet confirmed |
| Dependence on the parent's policy | It is described as independent, but both its funding and its business scope are subject to Bayer's decisions. Indeed, the description of its business areas was narrowed to neurology and ophthalmology in 2025 | Inference |
12. Glossary
- Pluripotent stem cell
- A stem cell that can turn into many kinds of cell. ES cells and iPS cells are the main examples.
- ES cell (embryonic stem cell)
- A pluripotent stem cell made from an embryo. The starting material for bemdaneprocel.
- iPS cell
- A stem cell made pluripotent by introducing genes into body cells such as skin cells. The starting material for OpCT-001.
- Allogeneic
- Using cells from another person. Immunosuppression is often needed to hold off rejection.
- Precursor cell
- A cell at a stage before becoming a fully mature cell. Expected to mature after transplantation.
- Sham surgery
- A surgical procedure in which no cells are actually transplanted. A control used to separate out expectation and the effect of the operation itself.
- RMAT
- Regenerative Medicine Advanced Therapy. An FDA programme that speeds up the development and review of regenerative medicine products. Designation does not mean approval.
- SAKIGAKE
- Japan's pioneering designation system for drugs and similar products (including regenerative medicine products). It can bring frequent consultation with the Ministry of Health, Labour and Welfare (MHLW) and priority review. It does not mean approval.
- Orphan drug designation
- A designation given to medicines for diseases with few patients; in the United States the guide is fewer than 200,000 patients. It is separate from approval.
- MDS-UPDRS Part III
- A rating scale that scores the motor symptoms of Parkinson's disease in a clinical examination.
13. References
- Bayer / BlueRock Bayer acquires BlueRock Therapeutics to build leading position in cell therapy (8 August 2019). Acquisition terms, the founding history and the iPS cell platform. https://www.bayer.com/media/en-us/bayer-acquires-bluerock-therapeutics-to-build-leading-position-in-cell-therapy/
- Bayer Bayer establishes Cell and Gene Therapy Platform to accelerate pharmaceutical innovation (2 December 2020). https://www.bayer.com/media/en-us/bayer-establishes-cell-and-gene-therapy-platform-to-accelerate-pharmaceutical-innovation/
- Bayer Bayer acquires Asklepios BioPharmaceutical to broaden innovation base in cell and gene therapy (26 October 2020). The AskBio acquisition. https://www.bayer.com/media/en-us/bayer-acquires-asklepios-biopharmaceutical-to-broaden-innovation-base-in-cell-and-gene-therapy/
- BlueRock / Bayer BlueRock Therapeutics announces FDA clearance of IND application for investigational iPSC-derived cell therapy OpCT-001 (3 September 2024). The relationship with FUJIFILM Cellular Dynamics and Opsis. https://www.bayer.com/media/en-us/bluerock-therapeutics-announces-fda-clearance-of-ind-application-for-investigational-ipsc-derived-cell-therapy-opct-001-for-the-treatment-of-primary-photoreceptor-diseases/
- BlueRock / Bayer BlueRock Therapeutics advances bemdaneprocel to registrational Phase III clinical trial (13 January 2025). https://www.bayer.com/media/en-us/bluerock-therapeutics-advances-investigational-cell-therapy-bemdaneprocel-for-treating-parkinsons-disease-to-registrational-phase-iii-clinical-trial/
- BlueRock / Bayer First Parkinson’s disease patient treated in BlueRock’s pivotal Phase III trial (22 September 2025). https://www.bayer.com/media/en-us/first-parkinsons-disease-patient-treated-in-bluerocks-pivotal-phase-iii-trial-of-investigational-cell-therapy-bemdaneprocel/
- BlueRock / Bayer bemdaneprocel receives Pioneering Regenerative Medical Product designation (SAKIGAKE) in Japan (17 December 2025). https://www.bayer.com/media/en-us/bluerock-therapeutics-investigative-cell-therapy-bemdaneprocel-for-treating-parkinsons-disease-receives-pioneering-regenerative-medical-product-designation-in-japan/
- BlueRock / Bayer FDA grants Orphan Drug Designation to OpCT-001 (22 January 2026). https://www.bayer.com/media/en-us/fda-grants-orphan-drug-designation-to-bluerock-therapeutics-investigational-cell-therapy-opct-001/
- BlueRock / Bayer Lemiretprocel (OpCT-001) granted orphan drug designations by FDA and EMA (30 September 2026). Also states Bayer's scale for FY2025. https://www.bayer.com/media/en-us/bluerock-therapeutics-investigational-cell-therapy-lemiretprocel-opct-001-for-primary-photoreceptor-diseases-granted-orphan-drug-designations-by-fda-and-ema/
- Peer-reviewed paper Tabar V et al., Phase I trial of hES cell-derived dopaminergic neurons for Parkinson’s disease. Nature 2025 (PMID 40240592). https://doi.org/10.1038/s41586-025-08845-y
- ClinicalTrials.gov NCT06944522 (exPDite-2: Phase 3 of bemdaneprocel). https://clinicaltrials.gov/study/NCT06944522
- ClinicalTrials.gov NCT06789445 (CLARICO: Phase 1/2a of OpCT-001). https://clinicaltrials.gov/study/NCT06789445
- ClinicalTrials.gov NCT04802733 (MSK-DA01, the Phase 1 trial of what is now bemdaneprocel). https://clinicaltrials.gov/study/NCT04802733
- ClinicalTrials.gov NCT05897957 (long-term follow-up of Phase 1 participants). https://clinicaltrials.gov/study/NCT05897957
- BlueRock Therapeutics Official website: pipeline (the logo was also obtained from this site). https://www.bluerocktx.com/the-science/pipeline/
14. Claim-to-source audit
Last updated 1 October 2026 / Troy Technical
Every figure in this article comes from official releases by Bayer and BlueRock, a peer-reviewed paper (Nature) and ClinicalTrials.gov registrations. No research-firm estimates or press-based figures have been used. Passages marked "Inference" are this article's interpretation of the primary sources, not statements made by the company. This article is not medical advice, does not recommend any particular treatment or product, and makes no comparison of its merits with other companies' products.