Berlin, Germany, and Cambridge, MA, USA听January 22, 2026听鈥� Bayer AG and its wholly owned, independently operated subsidiary 黑料大事记, a clinical stage cell therapy company, today announced that its investigational cell therapy, OpCT-001, has received Orphan Drug Designation from the U.S. FDA for treating retinitis pigmentosa (RP).听听

OpCT-001 is an investigational induced pluripotent stem cell (iPSC)-derived cell therapy being tested in a Phase 1/2a clinical study (CLARICO) for the treatment of primary photoreceptor diseases, which are a subgroup of inherited retinal disorders that include retinitis pigmentosa and cone-rod dystrophy. Retinitis pigmentosa is one of the most common types of inherited retinal diseases听characterized by the loss of rod and cone cells, known together as photoreceptor cells.听OpCT-001 aims to restore vision in people living with RP by replacing lost cells in the retina with new functional cells.听

鈥淩eceiving Orphan Drug Designation for retinitis pigmentosa is an important milestone for the OpCT-001 program,鈥� said Amit听Rakhit, MD, MBA,听BlueRock鈥檚听Chief Medical Officer. 鈥淲e believe that OpCT-001 has great promise as a potential therapeutic option for restoring vision in people living with retinitis pigmentosa and other primary photoreceptor diseases and look forward to continuing to work with the FDA on its clinical development.鈥�听

Orphan Drug Designation is given to drugs and biologics for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the U.S.鹿听听

鈥淭he FDA鈥檚 Orphan Drug Designation for OpCT-001 to treat retinitis pigmentosa听underscores the importance of developing urgently needed innovative therapies for patients living with听inherited retinal disorders,鈥� said Christian Rommel, Executive Vice President and Global Head of Research and Development of the Pharmaceuticals Division at Bayer. 鈥淭ogether with BlueRock, we are excited to be advancing the first-ever clinical trial for an iPSC-derived cell therapy in听primary photoreceptor diseases.鈥�听

OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.听

鹿;听;

About CLARICO听
CLARICO is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather听additional听safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups. Phase 1 will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4). Dose escalation in Phase 1 will be conducted using a standard 3+3 scheme in which a total of 12 to 24 legally blind participants (~3 to 6 per cohort) will receive escalating doses of OpCT-001 after acceptable safety signals with the prior lower听dose. Phase 2 is planned to enroll a maximum of 30 participants across 2 cohorts (Cohorts 5 and 6) to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either Cohort 5 or Cohort 6. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments. Further details of the trial can be found at听听(NCT06789445).听

About 黑料大事记
黑料大事记 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological and ophthalmic diseases. Two of our novel investigational cell therapies,听bemdaneprocel听(BRT-DA01) for the treatment of Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs.听Bemdaneprocel听has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase 3 clinical trial, exPDite-2. OpCT-001 has听Fast听Track designation from the FDA and is being tested in a Phase 1 clinical trial, CLARICO. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit听www.bluerocktx.com.听

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact听with听its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust,听reliability听and quality throughout the world. In fiscal 2024, the Group employed around听93,000 people听and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to听.听

Bayer Global Media Contact:
Dr. Imke Meyer, phone听+49 (214) 60001275
Email:听imke.meyer@bayer.com听

Kathryne Delves-Broughton, phone +33 6 02180163
Email:听kathryne.delvesbroughton@bayer.com听

BlueRock Media Contact:
Jeff Lockwood, phone +1 (617) 5106997
Email:听jlockwood@bluerocktx.com
Find more information at听.听

听

Forward-Looking Statements听黑料大事记
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be听identified听by the use of听forward-looking words such as 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渇orecast,鈥� 鈥渆stimate,鈥� 鈥減lan,鈥� and 鈥渋ntend,鈥� among others. These forward-looking statements are based on听BlueRock鈥檚听current expectations and actual results could differ materially. There are听a number of听factors that could cause actual events to differ materially from those听indicated听by such forward-looking statements. These factors include, but are not limited to, the initiation, timing, progress, activities, goals and reporting of results of any preclinical programs and clinical trials and research and development programs, the potential benefits, timing and future operation of the agreements with FUJIFILM Cellular Dynamics and听Opsis听Therapeutics, the ability to advance therapies into, and successfully initiate,听enroll听and complete clinical trials, the potential clinical utility of product candidates, the regulatory pathway of, and the timing or likelihood of any regulatory filings and approvals for, any product candidates, and the ability to, and extent of, potentially commercializing any product candidate, are forward looking. As with any pharmaceutical under development, there are significant risks in the development, regulatory听approval听and commercialization of new products. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement.听All of听BlueRock鈥檚听forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth听herein听speaks only as of the date hereof.

Forward-Looking Statements听
This release may听contain听forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks,听uncertainties听and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer鈥檚 public reports which are available on the Bayer website at听. The company assumes no liability whatsoever to update these forward-looking statements or to conform听them to future events or developments.听听

The post FDA grants Orphan Drug Designation to 黑料大事记鈥� investigational cell therapy OpCT-001 appeared first on 黑料大事记.

• Parkinson鈥檚 disease affects approximately听10 million people听worldwide听and听250,000听in Japan听

Berlin, Germany and Cambridge, MA, USA,听December 17,听2025听鈥撎鼴ayer AG and its wholly owned, independently operated subsidiary 黑料大事记, a clinical stage cell therapy company, today announced that the investigative cell therapy听bemdaneprocel, has received听Pioneering Regenerative Medical Product designation (SAKIGAKE)听from Japan鈥檚 Ministry of Health,听Labor听and Welfare (MHLW.) A drug candidate that receives Sakigake designation may be eligible for more frequent interaction with the MHLW to discuss the drug candidate鈥檚 development plan as well as eligibility for accelerated approval and priority听review, if听relevant criteria are met.鈥�听

鈥淲e are honored that听bemdaneprocel听has received Sakigake designation in Japan, recognizing the urgent need for new and innovative treatment options for people living with this debilitating condition,鈥� said Gabi Belfort, MD听PhD听and听Bemdaneprocel听Product Lead at 黑料大事记. 鈥淭his designation will allow us to work closely with Japan鈥檚 regulators to advance our therapy as efficiently as possible and bring new hope to people听living with Parkinson鈥檚 disease听and their families.鈥�听

Parkinson鈥檚 disease is the world鈥檚 second leading neurodegenerative disease, affecting approximately听10 million people听worldwide and听250,000听in Japan.听

鈥淩eceiving the Sakigake designation for听bemdaneprocel听highlights its potential to change how we approach Parkinson鈥檚 disease treatment,鈥� said Christian Rommel, PhD and Global Head of Research and Development for Bayer’s Pharmaceuticals Division. 鈥淥ur collaboration with the Japanese regulatory authority reflects听our听dedication to partnering with healthcare systems听and regulators听worldwide. Together, we aim to improve patient access to innovative therapies and make strides in enhancing patient outcomes.鈥濃��听听

About听bemdaneprocel听(BRT-DA01)
Bemdaneprocel听(BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson鈥檚 disease. These dopaminergic neuron precursors are derived from human embryonic pluripotent stem cells that continue developing into mature dopamine neurons after implantation. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson鈥檚 disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson鈥檚 disease and to potentially restore motor and non-motor function to patients. In听2021听bemdaneprocel听received Fast Track Designation and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. A pivotal Phase III听clinical听trial听(exPDite-2)听to assess the efficacy,听safety听and overall impact of听bemdaneprocel听compared to sham surgery control is currently enrolling participants.听Bemdaneprocel听has not been approved for treatment of any disease or medical condition by any health authority.听

About exPDite-2听
exPDite-2 is the first Phase III pivotal clinical trial for an investigational allogeneic pluripotent stem cell derived therapy to treat Parkinson鈥檚 disease. In a Phase I听study听with 12 participants,听bemdaneprocel听was well tolerated, with no serious adverse events related to drug听product听at 36 months post-surgery. In addition, encouraging trends were听observed听in secondary endpoints related to motor impairments at听36听months听post-surgery. Building on these results, exPDite-2 is a multicenter, double-blind trial that will assess the efficacy,听safety听and overall impact of听bemdaneprocel听compared to sham surgery control. The trial is designed to enroll approximately 102 participants with Parkinson鈥檚 disease听in US, Canada, and Australia. The primary endpoint of the听Phase III听study is听change听from baseline to week 78 in PD听motor听diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day. In addition, the trial will incorporate secondary endpoints designed to assess objective measures of movement, non-motor听symptoms, safety and tolerability, and instruments that capture activities of daily living and quality of life.听

About Parkinson鈥檚 disease
Parkinson鈥檚 disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person鈥檚 daily life. In PD, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement. Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks demanding. The prevalence of PD has doubled over the past 25 years. Today, more than听10 million people听worldwide are estimated to be living with PD. This makes it the world鈥檚 second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options are inadequate and lack holistic management of听symptoms听so there is an urgent need for new therapies.听

About 黑料大事记
黑料大事记 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological and ophthalmic diseases. Two of our novel investigational cell therapies,听bemdaneprocel听(BRT-DA01) for the treatment of Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs.听Bemdaneprocel听has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase III clinical trial, exPDite-2. OpCT-001 has Fast Track designation from the FDA and is being tested in a Phase听I听clinical trial, CLARICO听BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit听www.bluerocktx.com.听

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact听with听its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability,听and quality throughout the world. In fiscal 2024, the Group employed around听93,000 people听and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to听.听

Bayer contact听for media inquiries:听

Dr. Imke Meyer, phone听+49听214 60001275
Email:听imke.meyer@bayer.com
Find more information at听
Follow us on Facebook:听听听

BlueRock Contact for media听inquiries:听
Jeff Lockwood, phone +1 617 5106997
Email:听jlockwood@bluerocktx.com
Find information at听www.bluerocktx.com
Follow us on听LinkedIn:听听

Forward-Looking Statements听
This release may听contain听forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks,听uncertainties听and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer鈥檚 public reports which are available on the Bayer website at听. The company assumes no liability whatsoever to update these forward-looking statements or to conform听them to future events or developments.听

The post 黑料大事记 investigative cell therapy bemdaneprocel for treating Parkinson鈥檚 disease receives Pioneering Regenerative Medical Product designation in Japan appeared first on 黑料大事记.

Berlin, Germany and Cambridge, MA, USA, October 7, 2025 鈥� Bayer AG and its wholly owned, independently operated subsidiary 黑料大事记, a clinical stage cell therapy company, today announced positive 36-month data from exPDite, a Phase I clinical trial of bemdaneprocel, an investigational cell therapy for the treatment of Parkinson’s disease (PD). The data were presented on October 6th at the International Congress of Parkinson鈥檚 Disease and Movements Disorders.听

鈥淏emdaneprocel represents a new approach to restoring the dopamine inputs that are lost in Parkinson鈥檚, and leverages substantial advances in stem cell technology,鈥� said Claire Henchcliffe, MD, chair of the UC Irvine School of Medicine鈥檚 Department of Neurology at the University of California, Irvine and one of the study鈥檚 Principal Investigators. 鈥淭he new 3-year data is a critical next step to evaluate longer term safety. While there is a need for caution in interpreting the positive trends in clinical outcomes, initial signals are there, particularly in the higher dose cohort and the upcoming exPDite-2 clinical trial should shed further light on potential benefits.鈥�听

Bemdaneprocel鈥檚 safety profile at 36 months is consistent with earlier findings, demonstrating that it continues to be well tolerated by patients, with no adverse events reported related to the therapy or surgical procedure. F-Dopa imaging suggests that transplanted cells continue to survive and engraft in the brain after discontinuing immunosuppression therapy at 12 months as outlined in the study鈥檚 protocol. In addition, secondary clinical endpoints related to motor symptoms continue to show positive trends from baseline through the duration of follow-up, with more encouraging trends in the high dose cohort than those in the low dose cohort. These were assessed by the MDS-Unified Parkinson鈥檚 Disease Rating Scale Part II and III (MDS-UPDRS Part II & III) and the Parkinson鈥檚 disease Diary, tools used to assess Parkinson鈥檚 disease severity in motor symptoms.听

鈥淭he 36-month data from this Phase I study underscores our hope that bemdaneprocel could be a potentially meaningful and durable therapeutic option for treating people living with Parkinson鈥檚 disease,鈥� said Gabi Belfort, MD, PhD, Senior Vice President and Bemdaneprocel Product Lead at 黑料大事记. 鈥淲e are excited to share these data with the Parkinson鈥檚 disease community and are committed to advancing bemdaneprocel through the next stage of clinical testing.鈥�听

In the high dose cohort (n=7), the 36-month measurement of the effects of bemdaneprocel on motor symptoms using the MDS-UPDRS Part III measured in the 鈥淥FF鈥�-medication state, showed a mean reduction of 17.9 points compared with baseline. The low dose cohort (N=4) showed a mean decrease of 13.5 points. Both are considered to be clinically meaningful.1听

Using the PD Diary, which categorizes patients as being in the 鈥淥N鈥� state when their symptoms are well controlled and in the 鈥淥FF鈥� state when they experience a worsening of their symptoms, participants in the high dose cohort showed a mean increase of 1 hour in time spent in the 鈥淕ood ON鈥� state (without troublesome dyskinesias) compared with baseline after 36 months. Two participants in the high dose cohort did not submit a 36-month PD Diary. Time spent in the 鈥淥FF鈥� state showed a mean decrease of – 0.93 hours from baseline after 36 months. The participants in the low dose cohort showed a mean increase of 0.23 hours in the 鈥淕ood ON鈥� state time compared with baseline and a corresponding mean decrease of 鈥�1.15 hours in 鈥淥FF鈥� state time.听

In the high dose cohort, the 36-month measurement of the effects of bemdaneprocel on activities of daily living using the MDS-UPDRS Part II measured a mean reduction of -4.3 points compared with baseline. The low dose cohort showed a mean increase of 0.2 points. Participants in both cohorts continue in the long term Continued Evaluation Study.听

鈥淭he results for bemdaneprocel after 36 months mark another motivating milestone,鈥� said Christian Rommel, Global Head of Research and Development of the Pharmaceuticals Division at Bayer. 鈥淭hey reinforce our commitment to developing urgently needed new treatment options for Parkinson’s patients.鈥�听

About bemdaneprocel (BRT-DA01)
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson鈥檚 disease. These dopaminergic neuron precursors are derived from human embryonic pluripotent stem cells that continue developing into mature dopamine neurons after implantation. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson鈥檚 disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson鈥檚 disease and to potentially restore motor and non-motor function to patients. In 2021 bemdaneprocel received Fast Track Designation and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. A pivotal Phase III trial to assess the efficacy, safety and overall impact of bemdaneprocel compared to sham surgery control is currently enrolling participants. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority.听

About Parkinson鈥檚 disease
Parkinson鈥檚 disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person鈥檚 daily life. In PD, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement. Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks demanding. The prevalence of PD has doubled over the past 25 years. Today, more than 10 million people worldwide are estimated to be living with PD. This makes it the world鈥檚 second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options are inadequate and lack holistic management of symptoms so there is an urgent need for new therapies.听

About 黑料大事记
黑料大事记 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological and ophthalmic diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment of Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase III clinical trial, exPDite-2. OpCT-001 has Fast Track designation from the FDA and is being tested in a Phase I clinical trial, Clarico. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com.听

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to .听

Bayer contact for media inquiries:
Silke Lengemann, phone +49 173 8990997
Email: silke.lengemann@bayer.com听

BlueRock Contact for media inquiries:听
Jeff Lockwood, phone +1 617 5106997
Email: jlockwood@bluerocktx.com听

Find more information at
Follow us on Facebook: 听

Find information at www.bluerocktx.com
Follow us on LinkedIn: 听

Forward-Looking Statements听
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer鈥檚 public reports which are available on the Bayer website at . The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.听

1 Hauser RA, Gordon MF, Mizuno Y, Poewe W, Barone P, Schapira AH, Rascol O, Debieuvre C, Fr盲脽dorf M. Minimal clinically important difference in Parkinson’s disease as assessed in pivotal trials of pramipexole extended release. Parkinsons Dis. 2014;2014:467131. doi: 10.1155/2014/467131. Epub 2014 Apr 1. PMID: 24800101; PMCID: PMC3995302.听

The post 黑料大事记 reports positive 36-month results from Phase I trial of bemdaneprocel for treating Parkinson鈥檚 disease appeared first on 黑料大事记.

• Trial is a randomized, sham surgery-controlled double-blind study assessing efficacy and safety of bemdaneprocel in people living with Parkinson鈥檚 disease听

Cambridge, MA, USA September 22, 2025 鈥� 黑料大事记, a clinical-stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG, today announced that the first patient has received randomized treatment in the pivotal Phase III clinical trial, exPDite-2, of bemdaneprocel, an investigational cell therapy for Parkinson鈥檚 disease. 

鈥淧eople living with Parkinson鈥檚 disease urgently need new therapies that truly alter the course of the disease,鈥� said Amit Rakhit, MD, MBA, BlueRock鈥檚 Chief Development and Medical Officer. 鈥淭he initiation of the exPDite-2 trial represents a major step forward toward advancing bemdaneprocel and we are excited to build on the momentum of our earlier data to further develop it as a potentially transformative cell therapy aimed at restoring motor and non-motor function.鈥� 

exPDite-2 is the first Phase III pivotal clinical trial for an investigational allogeneic pluripotent stem cell derived therapy to treat Parkinson鈥檚 disease. In a Phase I study with 12 participants, bemdaneprocel was well tolerated, with no serious adverse events related to drug product at 24 months post-surgery. In addition, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. Building on these results, exPDite-2 is a multicenter, double-blind trial that will assess the efficacy, safety and overall impact of bemdaneprocel compared to sham surgery control. The trial is designed to enroll approximately 102 participants with Parkinson鈥檚 disease. The primary endpoint of the study is change from baseline to week 78 in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day. In addition, the trial will incorporate secondary endpoints designed to assess objective measures of movement, non-motor symptoms, safety and tolerability, and instruments that capture activities of daily living and quality of life. 

鈥淭he initiation of the expDite-2 Phase III trial marks a significant milestone in our commitment to transform the treatment landscape for Parkinson鈥檚 disease through innovative therapies,鈥� said Christian Rommel Ph.D., Head of Research and Development at Bayer鈥檚 Pharmaceuticals Division. 鈥淏emdaneprocel aims to sustainably restore lost physiologic function in the dopaminergic system impacted by the disease, ultimately to enhance the quality of life for patients.鈥� 

Depending upon the outcome, the results from this trial are intended to be part of a data package to support regulatory submissions for marketing authorization. 

About bemdaneprocel (BRT-DA01)听
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson鈥檚 disease. These dopaminergic neuron precursors are derived from human embryonic pluripotent stem cells that continue developing into mature dopamine neurons after implantation. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson鈥檚 disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson鈥檚 disease and to potentially restore motor and non-motor function to patients. In 2021 bemdaneprocel received Fast Track Designation and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Data from the Phase I trial鈥檚 12 participants presented at the 2024 International Congress of Parkinson鈥檚 Disease and Movement Disorders (MDS) demonstrated good tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. These participants continue in the long term Continued Evaluation Study. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority.听

About Parkinson鈥檚 disease听
Parkinson鈥檚 disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person鈥檚 daily life. In PD, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement. Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks demanding. The prevalence of PD has doubled over the past 25 years. Today, more than 10 million people worldwide are estimated to be living with PD. This makes it the听

world鈥檚 second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options are inadequate and lack the holistic management of symptoms so there is an urgent need for new therapies. 

About 黑料大事记听
黑料大事记 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological and ophthalmic diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment of Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase III clinical trial, exPDite-2. OpCT-001 has Fast Track designation from the FDA and is being tested in a Phase 1 clinical trial, Clarico. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com.听

About Bayer听
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to .听

Contact for media inquiries 黑料大事记: 

Jeff Lockwood, phone +1 (617) 5106997听
Email: jlockwood@bluerocktx.com听
Find more information at www.bluerocktx.com.听
听

Forward-Looking Statements听听
Certain statements in this press release are forward-looking which may be identified by the use of forward-looking words such as 鈥渁nticipate,鈥� 鈥渆xpect鈥�, 鈥渂elieve,鈥� 鈥渇orecast,鈥� 鈥渆stimate鈥� and 鈥渋ntend,鈥� among others. These forward-looking statements are based on BlueRock鈥檚 current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the outcomes from our clinical trials and ongoing FDA and other regulatory requirements, and interpretation of data by the FDA. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products, and uncertainty in the outcome of clinical trials cannot be avoided. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of BlueRock鈥檚 forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof.鈥�

The post First Parkinson鈥檚 disease patient treated in BlueRock鈥檚 pivotal Phase III trial of investigational cell therapy bemdaneprocel听 appeared first on 黑料大事记.

Berlin, Germany, and Cambridge, MA, USA July 8, 2025 鈥� Bayer AG and 黑料大事记, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today announced that the first patient received the investigational therapy in CLARICO, a Phase 1/2a clinical trial of OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. OpCT-001 is the first iPSC-derived investigational cell therapy to be clinically tested for treating primary photoreceptor (PR) diseases, which are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy.

鈥淭he initiation of the CLARICO trial represents a key milestone for the OpCT-001 program,鈥� said Amit Rakhit, MD, MBA, BlueRock鈥檚 Chief Development and Medical Officer. 鈥淲e believe OpCT-001 holds significant promise as a novel therapeutic approach for restoring vision in people living with primary photoreceptor diseases, and we look forward to assessing its safety and tolerability profile as we advance this important program in our pipeline.鈥�

CLARICO is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. Phase 1 will focus on safety and includes a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.

鈥淲e aim to transform treatment options for patients facing irreversible vision loss. OpCT-001, our investigational iPSC-derived cell therapy, has the potential to restore vision for individuals with primary photoreceptor disease,鈥� said Christian Rommel, Executive Vice President and Global Head of Research and Development of the Pharmaceuticals Division at Bayer. 鈥淲e are excited to announce the first patient in the CLARICO trial, the first-ever clinical trial for an iPSC-derived treatment in this field.鈥�

Primary photoreceptor diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. OpCT-001 aims to restore vision in patients with primary photoreceptor diseases by replacing degenerated cells in the retina with functional cells. Limited treatment options currently exist for treating primary photoreceptor diseases which affect an estimated 110,000鹿 people in the U.S.

OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

鹿https://www.ncbi.nlm.nih.gov/books/NBK519518/; https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1808442/; https://www.nidcd.nih.gov/health/usher-syndrome#b

About CLARICO
CLARICO is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups. Phase 1 will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4). Dose escalation in Phase 1 will be conducted using a standard 3+3 scheme in which a total of 12 to 24 legally blind participants (~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants in 2 cohorts (Cohorts 5 and 6) to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either Cohort 5 or Cohort 6. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments. Further details of the trial can be found at www.clinicaltrials.gov (NCT06789445).

About 黑料大事记
黑料大事记 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological and ophthalmic diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment of Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase 3 clinical trial, exPDite-2. OpCT-001 has Fast Track designation from the FDA and is being tested in a Phase 1 clinical trial, Clarico. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com.听听听听

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to .

Bayer Global Media Contact:
Dr. Imke Meyer, phone +49 (214) 60001275
Email: imke.meyer@bayer.com

BlueRock Media Contact:
Jeff Lockwood, phone +1 (617) 5106997
Email: jlockwood@bluerocktx.com

Find more information at .

Forward-Looking Statements 黑料大事记
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渇orecast,鈥� 鈥渆stimate,鈥� 鈥減lan,鈥� and 鈥渋ntend,鈥� among others. These forward-looking statements are based on BlueRock鈥檚 current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the initiation, timing, progress, activities, goals and reporting of results of any preclinical programs and clinical trials and research and development programs, the potential benefits, timing and future operation of the agreements with FUJIFILM Cellular Dynamics and Opsis Therapeutics, the ability to advance therapies into, and successfully initiate, enroll and complete clinical trials, the potential clinical utility of product candidates, the regulatory pathway of, and the timing or likelihood of any regulatory filings and approvals for, any product candidates, and the ability to, and extent of, potentially commercializing any product candidate, are forward looking. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of BlueRock鈥檚 forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof.

Forward-Looking Statements Bayer
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer鈥檚 public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

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The post Forging the future of cell therapy: Bayer and BlueRock鈥檚 unique journey appeared first on 黑料大事记.

Eye on the Cure Podcast | Episode 86: Lenore von Krusenstiern, MD, PhD

Dr. Lenore von Krusenstiern talks about Bluerock鈥檚 clinical trial for a photoreceptor replacement therapy for people with inherited retinal conditions such as retinitis pigmentosa and cone-rod dystrophy.

Podcast produced by Foundation Fighting Blindness, sponsored by 黑料大事记.

The post Eye on the Cure Podcast appeared first on 黑料大事记.

• Bemdaneprocel is the first investigational allogeneic pluripotent stem cell-derived cell therapy for Parkinson鈥檚 disease and the most clinically advanced cell therapy being investigated for treating the disease.鈥赌�&苍产蝉辫;

• BlueRock expects to initiate a Phase 3 clinical trial for bemdaneprocel in H1 2025.鈥赌�&苍产蝉辫;

Cambridge MA, USA, APRIL 16, 2025 – 黑料大事记, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today announced the publication of the 18-month data from its Phase 1 exPDite clinical trial for bemdaneprocel in the journal Nature.鈥赌�&苍产蝉辫;

鈥淭he concept of 鈥渞ebuilding鈥� brain networks that have been lost to disease is compelling,鈥� said Claire Henchcliffe, MD, chair of the UC Irvine School of Medicine鈥檚 Department of Neurology at the University of California, Irvine and one of the study鈥檚 Principal Investigators, 鈥淭he results of鈥痶his early phase clinical trial demonstrate鈥痶he promise of regenerative medicine and鈥痵hould provide hope for鈥疨arkinson鈥檚 disease鈥痯atients and their families.鈥濃��&苍产蝉辫;

The publication, which is available , confirms that 18 months following surgery, treatment with bemdaneprocel did not cause any serious adverse events related to cell therapy. In addition, positron emission tomography (PET) imaging techniques demonstrated sustained neuron cell engraftment in the trial鈥檚 high and low dose cohorts following discontinuation of immunosuppression after one year post-treatment. The publication also reports encouraging trends in secondary and exploratory subjective and objective endpoints related to motor function. These data were first presented in March 2024 at the Alzheimer鈥檚 and Parkinson鈥檚 Diseases Conference in Lisbon, Portugal.鈥��

鈥淐ell therapy is a potential new treatment option for individuals with Parkinson’s disease and our team is immensely proud of having bemdaneprocel鈥檚 18-month Phase 1 data published in such a prestigious journal as Nature,鈥� said Amit Rakhit, MD, MBA Head of Development and Chief Medical Officer at 黑料大事记. 鈥淥ur efforts are now focused on maintaining our forward momentum to initiate and enroll the Phase 3 trial as we advance bemdaneprocel鈥檚 development to this exciting next stage.鈥濃��&苍产蝉辫;

The Phase 3 trial, called exPDite-2, is expected to be initiated in the first half of 2025.鈥赌�&苍产蝉辫;

About bemdaneprocel (BRT-DA01)鈥��鈥�&苍产蝉辫;
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine-producing neurons that are lost in Parkinson鈥檚 disease. These dopaminergic neuron precursors are derived from human embryonic stem cells, developing into mature dopamine neurons after implantation. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson鈥檚 disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson鈥檚 disease and to potentially restore motor and non-motor function to patients. In 2021 bemdaneprocel received Fast Track designation followed in 2024 by a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.鈥� Data from the Phase 1 trial鈥檚 12 participants presented at the 2024 International Congress of Parkinson鈥檚 Disease and Movement Disorders (MDS) demonstrated good tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. These participants remain in a long-term Continued Evaluation Study. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority.鈥��赌�&苍产蝉辫;

About Parkinson鈥檚 disease鈥�鈥�&苍产蝉辫;
Parkinson鈥檚 disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person鈥檚 daily life. In PD, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement. Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks demanding. The prevalence of PD has doubled over the past 25 years. Today, more than 10 million people worldwide are estimated to be living with PD. This makes it the world鈥檚 second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options lack the holistic management of symptoms so new therapies are needed.鈥赌�&苍产蝉辫;

About 黑料大事记鈥���鈥�&苍产蝉辫;
黑料大事记 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological, ophthalmic, cardiovascular and immunological diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment of Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor disease are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is expected to begin a Phase 3 clinical trial in H1 2025. OpCT-001 is expected to begin Phase 1 clinical testing in H1 2025. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact-investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com.鈥����赌�&苍产蝉辫;

About Bayer鈥�&苍产蝉辫;
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to .鈥�&苍产蝉辫;

Contact for media inquiries:鈥�鈥�&苍产蝉辫;
Jeff Lockwood, phone +1 (617) 510 6997鈥�赌�&苍产蝉辫;
Email: jlockwood@bluerocktx.com鈥�赌�&苍产蝉辫;
Find information at www.bluerocktx.com鈥赌�&苍产蝉辫;

Follow us on LinkedIn: 鈥赌�&苍产蝉辫;

Forward-Looking Statements鈥�� 
Certain statements in this press release are forward-looking which may be identified by the use of forward-looking words such as 鈥渁nticipate,鈥� 鈥渆xpect鈥�, 鈥渂elieve,鈥� 鈥渇orecast,鈥� 鈥渆stimate鈥� and 鈥渋ntend,鈥� among others. These forward-looking statements are based on BlueRock鈥檚 current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the outcomes from our clinical trials and ongoing FDA and other regulatory requirements, and interpretation of data by the FDA. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products, and uncertainty in the outcome of clinical trials cannot be avoided. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of BlueRock鈥檚 forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof.鈥�&苍产蝉辫;

The post 黑料大事记 announces publication in Nature of 18-month data from Phase 1 clinical trial for bemdaneprocel, an investigational cell therapy for Parkinson鈥檚 disease appeared first on 黑料大事记.

• Fast Track designation is intended to facilitate the development and review of drug candidates that treat serious conditions and address an unmet medical need 

• Primary photoreceptor diseases are a subgroup of inherited retinal disorders that affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in children and adults 

Berlin, Germany, and Cambridge, MA, USA February 27, 2025 鈥� Bayer AG and 黑料大事记, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. OpCT-001 is the first iPSC-derived investigational cell therapy to be clinically tested for treating primary photoreceptor diseases, which are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy.  

The FDA鈥檚 Fast Track designation is intended to facilitate the development and review of drug candidates that treat serious conditions and address an unmet medical need. A drug candidate that receives Fast Track designation may be eligible for more frequent interaction with the FDA to discuss the drug candidate鈥檚 development plan as well as eligibility for accelerated approval and priority review, if relevant criteria are met. 

鈥淲e are thrilled to receive Fast Track designation from the FDA for OpCT-001,鈥� said Amit Rakhit, MD, MBA, BlueRock鈥檚 Chief Development and Medical Officer. 鈥淧eople living with primary photo receptor diseases need new treatments that have the potential to restore vision, and we look forward to working closely with the FDA to advance this program through clinical trials.鈥�  

Primary photoreceptor diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. OpCT-001 aims to restore vision in patients with primary photoreceptor diseases by replacing degenerated cells in the retina with functional cells. Limited treatment options currently exist for treating primary photoreceptor diseases which affect an estimated 110,000鹿 people in the U.S. 

鈥淩eceiving FDA Fast Track designation for OpCT-001 demonstrates the urgent need for innovative treatments in the field of primary photoreceptor diseases,鈥� said Christian Rommel, Executive Vice President, Global Head of Research and Development, and Member of the Pharmaceuticals Leadership Team at Bayer. 鈥淲e are proud to deliver on our commitment to advancing groundbreaking cell therapies by seeking to develop the first iPSC-derived investigational cell therapy for patients facing devastating consequences from primary photoreceptor diseases.鈥� 

OpCT-001 was exclusively licensed from FUJIFILM Cellular Dynamics and Opsis Therapeutics in January 2024 as part of the strategic R&D and clinical manufacturing collaboration between 黑料大事记, FUJIFILM Cellular Dynamics, and Opsis Therapeutics forged in 2021. As part of the collaboration, FUJIFILM Cellular Dynamics supported 黑料大事记 via research, development and the execution of critical IND-enabling activities including the clinical manufacturing of OpCT-001. 

OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. 

鹿https://www.ncbi.nlm.nih.gov/books/NBK519518/; https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1808442/; https://www.nidcd.nih.gov/health/usher-syndrome#b 

About 黑料大事记听
黑料大事记 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological, ophthalmic, cardiovascular and immunological diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor disease are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the U.S. FDA (Food and Drug Administration); has completed Phase 1 clinical trial and is progressing to the next stage of clinical development. OpCT-001 has been cleared by the FDA to begin Phase 1 clinical testing. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com.听

About Bayer 
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to . 

Bayer Global Media Contact: 
Dr. Imke Meyer, phone +49 (214) 60001275 
Email: imke.meyer@bayer.com 

BlueRock Media Contact:  
Jeff Lockwood, phone +1 (617) 5106997 
Email: jlockwood@bluerocktx.com 

Find more information at . 

Forward-Looking Statements 黑料大事记 
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渇orecast,鈥� 鈥渆stimate,鈥� 鈥減lan,鈥� and 鈥渋ntend,鈥� among others. These forward-looking statements are based on BlueRock鈥檚 current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the initiation, timing, progress, activities, goals and reporting of results of any preclinical programs and clinical trials and research and development programs, the potential benefits, timing and future operation of the agreements with FUJIFILM Cellular Dynamics and Opsis Therapeutics, the ability to advance therapies into, and successfully initiate, enroll and complete clinical trials, the potential clinical utility of product candidates, the regulatory pathway of, and the timing or likelihood of any regulatory filings and approvals for, any product candidates, and the ability to, and extent of, potentially commercializing any product candidate, are forward looking. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of BlueRock鈥檚 forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof. 

Forward-Looking Statements Bayer 
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer鈥檚 public reports which are available on the Bayer website at . The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.  

The post 黑料大事记 receives FDA Fast Track Designation for OpCT-001 for the treatment of primary photoreceptor diseases听 appeared first on 黑料大事记.

Berlin, Germany, and Cambridge, MA, USA, January 13, 2025 鈥� Bayer AG and 黑料大事记, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today announced plans to initiate a Phase III clinical trial for bemdaneprocel, its investigational cell therapy for Parkinson鈥檚 disease. The registrational trial, named exPDite-2, is expected to begin in the first half of 2025 and will represent a significant milestone in the development of allogeneic cell-based therapies for neurodegenerative disorders.听

鈥淲e are thrilled to take this critical step in our development program towards further investigating a potential new therapeutic option for people living with Parkinson鈥檚 disease,鈥� said Amit Rakhit, MD, MBA, Chief Development and Medical Officer at 黑料大事记. 鈥渆xPDite-2 is the first registrational Phase III clinical trial for an investigational pluripotent stem cell derived therapy in Parkinson鈥檚 disease and we look forward to working closely with clinical investigators and the Parkinson鈥檚 disease community as we initiate this trial.鈥�听

In a Phase I study with 12 participants, bemdaneprocel demonstrated tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. Building on these results, exPDite-2 is a double-blind trial that will assess the efficacy, safety and overall impact of bemdaneprocel compared to a sham surgery control. The trial is designed to enroll approximately 102 participants with moderate Parkinson鈥檚 disease. The primary endpoint of the study is change from baseline to week 78 in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day. In addition, the trial will incorporate secondary endpoints designed to capture objective measures of movement, safety and tolerability, and instruments that capture activities of daily living and quality of life.听

The advancement to this registrational trial follows discussions with the U.S. Food and Drug Administration (FDA) under the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the FDA in May 2024.听

鈥淲ith the planned initiation of the Phase III clinical trial, we are committed to bringing bemdaneprocel faster to patients in need,鈥� said Christian Rommel, Member of the Executive Committee of Bayer鈥檚 Pharmaceuticals Division, and Global Head of Research and Development. 鈥淚t represents a significant milestone in our efforts to advance our cell and gene therapy pipeline and deliver on our ambition to be an industry leader in this space.鈥�听

Depending upon the outcome, the results from this trial are intended to be part of a robust data package to support regulatory submissions for marketing authorization.听

About bemdaneprocel (BRT-DA01)听
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson鈥檚 disease. These dopaminergic neuron precursors are derived from pluripotent stem cells that are human embryonic stem cells developing into mature dopamine neurons after implantation. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson鈥檚 disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson鈥檚 disease and to potentially restore motor and non-motor function to patients. In 2021 bemdaneprocel received Fast Track Designation and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Data from the Phase I trial鈥檚 12 participants presented at the 2024 International Congress of Parkinson鈥檚 Disease and Movement Disorders (MDS) demonstrated good tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. These participants continue in the long term Continued Evaluation Study. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority.听

About Parkinson鈥檚 disease
Parkinson鈥檚 disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person鈥檚 daily life. In PD, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement. Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks demanding. The prevalence of PD has doubled over the past 25 years. Today, more than 10 million people worldwide are estimated to be living with PD. This makes it the world鈥檚 second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options lack the holistic management of symptoms so new therapies are needed.听

About 黑料大事记
黑料大事记 is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological, ophthalmic, cardiovascular and immunological diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment of Parkinson鈥檚 disease and OpCT-001 for the treatment of primary photoreceptor disease are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is expected to begin a Phase III clinical trial in H1 2025. OpCT-001 is expected to begin Phase I clinical testing in H1 2025. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com.听

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, 鈥淗ealth for all, Hunger for none,鈥� the company鈥檚 products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to .听

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Email: imke.meyer@bayer.com听

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Bayer Investor Relations Team, phone +49 214 30-72704
Email: ir@bayer.com听听

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Contact for media inquiries 黑料大事记:
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This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer鈥檚 public reports which are available on the Bayer website at . The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.听听

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