FUJIFILM Corp.

CAMBRIDGE, MA USA I September 3, 2024 I BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG, today announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. “We are pleased with the immense progress we have made to date and thrilled that the FDA has cleared our IND application to initiate clinical testing for OpCT-001,” said Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics. “We believe that OpCT-001 has potential to restore vision in people living with primary photoreceptor diseases and look forward to working with the ophthalmology community in initiating our Phase 1/2a clinical study.” OpCT-001 is the first investigational iPSC-derived cell therapy to be clinically evaluated for the treatment of primary photoreceptor diseases. Initiation activities to support a Phase 1/2a study are underway. The Phase 1/2a study is designed as a first-in-human study to evaluate the safety and tolerability of subretinal administration of OpCT-001 in people with primary photoreceptor diseases as well as evaluate the effect of OpCT-001 on retinal structure, visual function and functional vision. The study will assess several dose levels of OpCT-001 and is expected to enroll participants in sites across the U.S. Primary photoreceptor diseases are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy. These diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. Primary photoreceptor diseases affect an estimated 110,000¹ people in the U.S. and only limited treatment options exist for this population. OpCT-001 aims to restore vision loss caused by these diseases by replacing degenerated cells in the retina with functional cells. 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 BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics forged in 2021. As part of the collaboration, FUJIFILM Cellular Dynamics supported BlueRock Therapeutics via research, development and the execution of critical IND-enabling activities including the clinical manufacturing of OpCT-001 at their cGMP² facility in Madison, Wisconsin. 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. ¹https://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 ²Current good manufacturing practices are regulations enforced by the FDA. About BlueRock Therapeutics LP BlueRock Therapeutics LP 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’s 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. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its 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 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com . Find more information at https://pharma.bayer.com Follow us on Facebook: http://www.facebook.com/bayer SOURCE: Bluerock Therapeutics

DUBLIN--( BUSINESS WIRE )--The "Induced Pluripotent Stem Cell (iPSC) Industry Report - Market Size, Trends, and Forecasts, 2024" report has been added to ResearchAndMarkets.com's offering. This global strategic report reveals all major market competitors worldwide, including their core technologies, strategic partnerships, and products under development. It covers the current status of iPSC research, biomedical applications, manufacturing technologies, patents, and funding events, as well as all known trials for the development of iPSC-derived cell therapeutics worldwide. Importantly, it profiles leading market competitors worldwide and presents a comprehensive market size breakdown for iPSCs by Application, Technology, Cell Type, and Geography (North America, Europe, Asia/Pacific, and Rest of World). It also presents total market size figures with projected growth rates through 2030. Since the discovery of induced pluripotent stem cell (iPSC) technology in 2006, significant progress has been made in stem cell biology and regenerative medicine. New pathological mechanisms have been identified and explained, new drugs identified by iPSC screens are in the pipeline, and the first clinical trials employing human iPSC-derived cell types have been initiated. iPSCs can be used to explore the causes of disease onset and progression, create and test new drugs and therapies, and treat previously incurable diseases. Today, methods of commercializing induced pluripotent stem cells (iPSCs) include: Cellular Therapy: iPSCs are being explored in a diverse range of cell therapy applications for the purpose of reversing injury or disease. Disease Modelling: By generating iPSCs from patients with disorders of interest and differentiating them into disease-specific cells, iPSCs can effectively create disease models "in a dish". Drug Development and Discovery: iPSCs have the potential to transform drug discovery by providing physiologically relevant cells for compound identification, target validation, compound screening, and tool discovery. Personalized Medicine: The use of techniques such as CRISPR enable precise, directed creation of knock-outs and knock-ins (including single base changes) in many cell types. Pairing iPSCs with genome editing technologies is adding a new dimension to personalized medicine. Toxicology Testing: iPSCs can be used for toxicology screening, which is the use of iPSCs or their derivatives (tissue-specific cells) to assess the safety of compounds or drugs within living cells. Tissue Engineering: iPSCs can be seeded onto scaffolds made from biocompatible materials. These scaffolds mimic the structure and properties of the target tissue and can provide a supportive environment for cell growth and differentiation. Organoid Production: iPS cells can be coaxed to self-organize into 3D structures called organoids, which mimic the structure and function of organs. Organoids can be used for studying organ development, modeling diseases, and testing drugs. Gene Editing: iPS cells can be genetically modified using techniques like CRISPR-Cas9 to correct disease-causing mutations or introduce specific genetic changes. These edited iPS cells can then be differentiated into the desired cell type for transplantation or disease modeling. Research Tools: iPSCs and iPSC-derived cell types are being widely used within a diverse range of basic and applied research applications. Stem Cell Banking: iPSC repositories provide researchers with the opportunity to investigate a diverse range of conditions using iPSC-derived cell types produced from both healthy and diseased donors. Cultured Meat Production: iPSCs are being utilized in clean meat production by serving as the cellular foundation for the creation of lab-grown meat. 3D Bioprinting: iPSCs can be directed to differentiate into cell types of interest, such as skin, heart, or liver cells, which are then incorporated into bioinks. Wildlife Conservation and De-extinction Projects: iPSCs are being used in wildlife conservation and de-extinction projects. For example, Colossal Biosciences is using iPSC technology in an effort to achieve woolly mammoth de-extinction. Key Topics Covered: 1. REPORT OVERVIEW 2. INTRODUCTION 3. CURRENT STATUS OF IPSC INDUSTRY 3.1 Progress made in Autologous Cell Therapy using iPSCs 3.2 Allogeneic iPSC-based Cell Therapies 3.3 Share of iPSC-based Research within the Overall Stem Cell Industry 3.4 Major Focus Areas of iPSC Companies 3.5 Commercially Available iPSC-derived Cell Types 3.6 Relative use of iPSC-derived Cell Types in Toxicology Testing Assays 3.7 iPSC-derived Cell Types used in Clinical Trials 3.8 Currently Available iPSC Technologies Nucleofector Technology Opti-ox Technology MOGRIFY Technology Transcription Factor-based iPSC Differentiation Technology Flowfect Technology Technology for Mass Production of Platelets SynFire Technology 4. HISTORY OF INDUCED PLURIPOTENT STEM CELLS (IPSCS) 5. RESEARCH PUBLICATIONS ON IPSCS 6. IPSC: PATENT LANDSCAPE ANALYSIS 6.1 iPSC Patent Applications by Jurisdiction 6.2 iPSC Patent Applicants 6.3 Inventors of iPSC Patents 6.4 iPSC Patent Owners 6.5 Legal Status of iPSC Patents 7. IPSC: CLINICAL TRIAL LANDSCAPE 7.1 Number of iPSC Clinical Trials 7.2 Recruitment Status of iPSC Clinical Trials 7.3 iPSC Clinical Trials Study Designs 7.4 Therapeutic & Non-Therapeutic iPSC Clinical Trials 7.5 iPSC-based Trials by Phase of Study 7.6 iPSC Clinical Trials by Funder Type 7.7 Geographic Distribution of iPSC-based Clinical Trials 7.8 Promising iPSC Product Candidates CYP-001, CYP-004 & CYP-006 from Cynata Therapeutics BioVAT-HF from Repairon GmbH HS-001 from Heartseed CNTY-101 from Century Therapeutics FT-576 & FT-819 from Fate Therapeutics RPE from National Eye Institute QN-019a from Qihan Biotech iPSC-CL from Heartworks, Inc. 7.9 Companies having Preclinical iPSC Assets Aspen Neuroscience Ryne Biotech Bluerock Therapeutics Vita Therapeutics Hopstem Biotechnology Res Nova Bio, Inc. Cytovia Therapeutics Hebecell Corporation Sana Biotechnology SCG Cell Therapy Pte Cytomed Shoreline Biosciences Neukio Biotherapeutics Exacis Biotherapeutics CellOrigin Biotech 8. M&A, COLLABORATIONS & FUNDING ACTIVITIES IN IPSC SECTOR 8.1 Mergers and Acquisitions (M&A) Sector Century Therapeutics & Clade Therapeutics Evotech & Rigenerand Fujifilm Corporation & Atara Biotherapeutics Catalent & RheinCell Therapeutics Axol Biosciences & Censo Biotechnologies Bayer AG & Bluerock Therapeutics Pluriomix & Axiogenesis 8.2 Partnership/Collaboration & Licensing Deals in iPSC Sector Shinobi Therapeutics & Panasonic SCG Cell Therapy and A*STAR Charles River Laboratories & Pluristyx, Inc. Pluristyx, Inc. & National Resilience, Inc. University of Texas & GeneCure Heartseed, Inc. & Undisclosed Biotech Bluerock Therapeutics & Bit.bio Applied Stem Cell, Inc. & CIRM Resolution Therapeutics & OmniaBio, Inc. REPROCELL, Inc. & CIRM REPROCELL, Inc. & BioBridge Global Elevate Bio & CIRM Evotec & Sernova Evotec & Almiral Quell Therapeutics & Cellistic MDimmune & YiPSCELL Edigene & Neukio Biotherapeutics Matricelf & Ramot Evotec & Boehringer Ingelheim Pluristyx, Pancella & Implant Therapeutics Century Therapeutics & Bristol Myers Squibb Fujifilm Cellular Dynamics & Pheno Vista Biosciences Metrion Biosciences & Bioqube Ventures Cytovia Therapeutics & Cellectis Exacis Biotherapeutics & CCRM Cynata Therapeutics & Fujifilm Corporation Bone Therapeutics & Implant Therapeutics REPROCELL & TEXCELL Jacobio & Herbecell NeuCyte & KIF1A.ORG Kite & Shoreline Biosciences Neuropath Therapeutics & Hopstem Biotechnology Allele Biotech & Cellatoz Bluerock Therapeutics, Fujifilm Cellular Dynamics & Opsis Therapeutics Newcells & Takeda Biocentriq & Kytopen Fujifilm Cellular Dynamics & Sana Biotechnology Evotec & Medical Center Hamburg-Eppdorf (UKE) NeuCyte & Seaver Autism Center for Research and Treatment Cytovia Therapeutics & National Cancer Institute Mogrify & MRC Laboratory of Molecular Biology 8.3 Venture Capital Funding in iPSC Sector Asgard Therapeutics Kenai Therapeutics Pluristyx Fujifilm Cellular Dynamics Mogrify Ltd. Heartseed, Inc. Elevate Bio Aspen Neurosciences Axol Biosciences Thyas, Co. Ltd Synthego Cellino Biotech, Inc Curi Bio Ncardia Evotec SE bit.bio Clade Therapeutics Shoreline Biosciences Kytopen Cytovia Therapeutics & CytoLynx TreeFrog Therapeutics HebeCell Corporation Neukio Biotherapeutics Stemson Therapeutics Vita Therapeutics Century Therapeutics Heartseed Mogrify Metrion Biosciences Elevate Bio Vita Therapeutics 9. GENERATION OF INDUCED PLURIPOTENT STEM CELLS (IPSCS) 9.1 OSKM Cocktail 9.2 Pluripotency-Associated Transcription Factors and their Functions 9.3 Delivery of Reprogramming Factors 9.3.1 Integrating Systems 9.3.2 Non-Integrative Delivery Systems 9.3.3 Comparison of Delivery Methods 9.4 Genome Editing Technologies in iPSC Generation 9.5 Available iPSC Lines and their applications 10. HUMAN IPSC BANKING 10.1 Major Biobanks Storing iPSCs & iPSC Lines 10.1.1 RIKEN 10.1.2 WiCell 10.1.3 Fujifilm Cellular Dynamics, Inc. 10.1.4 Sampled 10.1.5 Coriell Institute for Medical Research 10.1.6 European Bank for Induced Pluripotent Stem Cells (EBiSC) 10.2 Cell Sources for iPSC Banks 10.3 Reprogramming Methods in iPSC Banks 10.4 Ownership and Investments made in iPSC Banks 11. BIOMEDICAL APPLICATIONS OF IPSCs 11.1 iPSCs in Basic Research 11.2 Applications of iPSCs in Drug Discovery 11.3 Applications of iPSCs in Toxicology Studies 11.4 Applications of iPSCs in Disease Modeling 11.5 Applications of iPSCs in Cell-Based Therapies 11.6 Other Novel Applications of iPSCs 12. MARKET ANALYSIS 12.1 Global Market for iPSCs by Geography 12.2 Global Market for iPSCs by Technology 12.3 Global Market for iPSCs by Biomedical Application 12.4 Global Market for iPSCs by Derived Cell Type 12.5 Market Drivers 12.5.1 Current Drivers Impacting the iPSC Market Place 12.6 Market Restraints 13. COMPANY PROFILES For more information about this report visit https://www.researchandmarkets.com/r/pf57qx About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

DALLAS, June 27, 2024 /PRNewswire/ -- Jacobs (NYSE:J) was selected by FUJIFILM Diosynth Biotechnologies to support delivery of $1.2 billion expansion of an end-to-end, large-scale cell culture contract manufacturing site in Holly Springs, North Carolina. Jacobs was originally selected to support the construction for the FUJIFILM Diosynth Biotechnologies' Holly Springs site, which broke ground in 2021. Jacobs will provide engineering, procurement and construction management services for the site's latest expansion. "This expansion of the project at Holly Springs is testament to the relationship of trust and accountability Jacobs and FUJIFILM Diosynth Biotechnologies have built," said Jacobs Senior Vice President Koti Vadlamudi. "Together we'll continue to leverage digital innovation and modular design so that medicines and therapies are delivered with quality, flexibility, speed and consistency—now at an even larger scale." The expansion will leverage the existing engineering and facility designs, with an additional 8 x 20,000 liter (L) cell culture bioreactors and approximately 400,000 square feet of manufacturing space by 2028, to the already planned 8 x 20,000 L for bulk drug substance as part of the initial investment. The first phase of construction is planned for completion in 2025. "Once complete, the site will be one of the largest, end-to-end cell culture contract manufacturing facilities in North America," said FUJIFILM Diosynth Biotechnologies COO and Executive Vice President of Operations Kenneth Bilenberg. "Leveraging our KojoXTM philosophy, the project will significantly increase FUJIFILM Diosynth Biotechnologies' manufacturing capacity for biologic therapeutics and medicines for patients globally. The site will provide drug substance manufacturing, automated fill-finish and assembly, packaging and labeling services for global biopharmaceutical customers." At Jacobs, we're challenging today to reinvent tomorrow by solving the world's most critical problems for thriving cities, resilient environments, mission-critical outcomes, operational advancement, scientific discovery and cutting-edge manufacturing, turning abstract ideas into realities that transform the world for good. With approximately $16 billion in annual revenue and a talent force of more than 60,000, Jacobs provides a full spectrum of professional services including consulting, technical, scientific and project delivery for the government and private sector. Visit jacobs.com and connect with Jacobs on Facebook, Instagram, LinkedIn and X. Certain statements contained in this press release constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that do not directly relate to any historical or current fact. When used herein, words such as "expects," "anticipates," "believes," "seeks," "estimates," "plans," "intends," "future," "will," "would," "could," "can," "may," and similar words are intended to identify forward-looking statements. We base these forward-looking statements on management's current estimates and expectations, as well as currently available competitive, financial and economic data. Forward-looking statements, however, are inherently uncertain. There are a variety of factors that could cause business results to differ materially from our forward-looking statements including, but not limited to, our plans to spin off and merge with Amentum our Critical Missions Solutions business and a portion of our Divergent Solutions business in a proposed transaction that is intended to be tax-free to stockholders for U.S. federal income tax purposes, the timing of the award of projects and funding and potential changes to the amounts provided for under the Infrastructure Investment and Jobs Act and other legislation related to governmental spending, as well as general economic conditions, including inflation and the actions taken by monetary authorities in response to inflation, changes in interest rates and foreign currency exchange rates, changes in capital markets, the possibility of a recession or economic downturn, geopolitical events and conflicts, and the impact of any future pandemic or infectious disease outbreak, including the related reaction of governments on global and regional market conditions, among others. For a description of some additional factors that may occur that could cause actual results to differ from our forward-looking statements, see the discussions contained under Item 1 - Business; Item 1A - Risk Factors; Item 3 - Legal Proceedings; and Item 7 - Management's Discussion and Analysis of Financial Condition and Results of Operations in our most recently filed Annual Report on Form 10-K, and Item 2 - Management's Discussion and Analysis of Financial Condition and Results of Operations; Item 1 - Legal Proceedings; and Item 1A - Risk Factors in our most recently filed Quarterly Report on Form 10-Q, as well as the company's other filings with the Securities and Exchange Commission. The company is not under any duty to update any of the forward-looking statements after the date of this press release to conform to actual results, except as required by applicable law. For press/media inquiries: [email protected] SOURCE Jacobs