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January Monthly Edition-2026

CIO Bulletin

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BlueRock Therapeutics – Rewriting the Rules of Medicine by Turning Stem Cell Science into Real, Restorative Healing

Every breakthrough in medicine starts with a question that challenges what is accepted as “normal.” At BlueRock Therapeutics, those questions are not theoretical, they are deeply human. What if damaged cells could be replaced instead of managed? What if patients could move beyond lifelong symptom control and experience real restoration? These are not distant ambitions inside BlueRock’s labs. They are the driving force behind a company that is quietly reshaping how diseases are treated. This bold yet grounded pursuit of healing places BlueRock Therapeutics at the center of the Stem Cell Innovation.

Rather than chasing incremental change, BlueRock focuses on rewriting the fundamentals of treatment. Its work reflects patience, creativity, and a deep respect for the lives waiting on better answers.

BlueRock Therapeutics and the Art of Cell Therapy

Founded to explore the untapped potential of regenerative medicine, BlueRock Therapeutics is a clinical-stage cell therapy company dedicated to developing treatments that replace cells lost or damaged by disease. The company operates as a wholly owned, independently run subsidiary of Bayer AG, forming a cornerstone of Bayer’s Cell and Gene Therapy platform while retaining its own scientific identity and creative freedom.

BlueRock approaches science much like an artist approaches a blank canvas, with imagination, discipline, and the intention to create something lasting. Its mission is clear: to discover and develop cell therapies that fundamentally change how diseases are treated and improve the lives of patients worldwide.

At its core, BlueRock believes the future of medicine lies not in managing decline, but in restoring function. This belief guides every research decision, clinical trial, and partnership the company undertakes.

Harnessing the Power of Cells to Treat Disease at the Source

Traditional medicine often focuses on managing symptoms over time. BlueRock is working toward a different outcome. Its science is built on the idea that many diseases result from the loss or damage of specific cell types, and that replacing those cells could restore normal function.

To achieve this, BlueRock uses pluripotent stem cells, which have the unique ability to become many different cell types. The company’s primary focus is on induced pluripotent stem cells, or iPSCs. These cells are created by reprogramming fully developed adult donor cells back into a flexible, “blank canvas” state. From there, they can be guided to become highly specific cells needed to repair damaged tissue.

This approach allows BlueRock to design therapies with precision, consistency, and scalability, qualities essential for real-world clinical impact.

In certain cases, cell replacement alone is not enough. BlueRock’s cell-plus-gene platform allows scientists to enhance stem cells using gene editing technologies when necessary. This enables cells to function more effectively, remain stable in the body, or deliver therapeutic benefits in a controlled manner.

By combining cellular therapy with genetic engineering, BlueRock expands what regenerative medicine can achieve. This integrated approach reflects the company’s commitment to innovation without compromising safety or scientific rigor.

Manufacturing Innovation at Clinical Scale

Scientific discovery means little without the ability to deliver therapies reliably. BlueRock’s manufacturing teams play a critical role in translating research into clinical reality. They produce stable, high-quality cell supplies for both early- and late-stage clinical trials.

Through close collaboration with Bayer, BlueRock is also preparing for large-scale manufacturing. This focus ensures that promising therapies can move beyond the lab and reach patients globally, a challenge many cell therapy companies struggle to overcome.

Transforming Treatment in Neurology

One of BlueRock’s most advanced programs focuses on neurology, particularly Parkinson’s disease. Parkinson’s is caused by the progressive loss of dopamine-producing neurons in the brain, leading to motor and cognitive challenges that worsen over time. Despite affecting millions worldwide, treatment options have changed little in decades.

BlueRock’s investigative therapy, bemdaneprocel, aims to replace the lost dopamine-producing cells themselves. Derived from human embryonic stem cells, this therapy represents a potential shift from symptom management to functional restoration. Currently in clinical development, bemdaneprocel embodies BlueRock’s vision of addressing disease at its root rather than its surface.

Restoring Sight Through Ophthalmology Innovation

BlueRock is also advancing therapies in ophthalmology, focusing on inherited retinal diseases that cause vision loss at a young age. These conditions result from the loss of light-sensing photoreceptor cells, which the body cannot naturally regenerate.

Through its ophthalmology program, OpCT-001, BlueRock is investigating pluripotent stem cell-based therapies designed to replace these lost cells. Developed in partnership with Opsis Therapeutics and FUJIFILM Cellular Dynamics, this program targets primary photoreceptor diseases and represents hope for patients facing irreversible vision loss.

Collaboration as a Catalyst for Progress

BlueRock recognizes that creating transformative cell therapies is a collective effort. The company actively collaborates with organizations that share its ambition and values. Partnerships with Opsis Therapeutics, Editas Medicine, and Senti Biosciences allow BlueRock to combine clinical expertise, gene editing technologies, and advanced gene circuits to push the boundaries of what cell therapy can achieve.

These collaborations strengthen BlueRock’s scientific platform while accelerating development timelines and expanding therapeutic possibilities.

Scripting a New Chapter in Medicine

BlueRock Therapeutics is not chasing headlines, it is building foundations. Through disciplined science, thoughtful collaboration, and a clear focus on patients, the company is turning long-standing medical questions into real therapeutic pathways.

Its work reflects a future where diseases are treated at their source, where lost cells can be replaced, and where patients are offered not just relief, but renewal. That vision, combined with measurable clinical progress, makes BlueRock Therapeutics a defining force in regenerative medicine.

About | Seth Ettenberg, Ph.D.

Seth Ettenberg, Ph.D., is the President and CEO of BlueRock Therapeutics. Seth served as Chief Scientific Officer at BlueRock from 2020–2021. Prior to joining BlueRock, Seth was a founding member of the Unum Therapeutics team, where he served as Chief Scientific Officer. Previously, Seth headed the Cambridge Site of Novartis’ Oncology Biotherapeutics, leading strategy, innovation and implementation of drug development projects for Novartis Institutes for Biomedical Research (NIBR). In addition, Seth was responsible for building and leading the Novartis Cell and Gene Therapy research team in collaboration with the University of Pennsylvania to develop chimeric antigen receptor T-cell therapeutics. Seth received his formal scientific training at the Genetics Branch of the National Cancer Institute. Seth is a member of the Board of Directors for Kano Therapeutics and the 2026 Board of Directors of the Alliance for Regenerative Medicine.

“We are driven to tackle complex science for the benefit of patients. Part of what makes BlueRock work is our ability to collaborate as a team of creative, passionate colleagues.”

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