FUJIFILM Cellular Dynamics and Opsis Therapeutics Announce License with BlueRock Therapeutics for iPSC-derived Cell Therapy Candidate

MADISON, Wis., January 23, 2024 – FUJIFILM Cellular Dynamics, Inc., a leading global developer and manufacturer of human induced pluripotent stem cells (iPSC), and Opsis Therapeutics, LLC, a joint venture of FUJIFILM Cellular Dynamics and co-founder David Gamm, M.D., Ph.D., focusing on developing cell therapies for patients with ocular diseases, today announced an agreement granting BlueRock Therapeutics (Cambridge, Mass.) an exclusive, worldwide license to develop and commercialize an iPSC-derived cell therapy candidate for the treatment of human retinal diseases. BlueRock Therapeutics, a clinical stage cell therapy company and wholly owned independently operated subsidiary of Bayer AG, exercised its option to exclusively license OpCT-001, an iPSC-derived photoreceptor cell therapy for inherited retinal disorders.

Following the option exercised by BlueRock Therapeutics, FUJIFILM Cellular Dynamics and Opsis Therapeutics received an undisclosed license fee and are eligible to receive payments upon achievement of certain development and commercial milestones.

OpCT-001 is the first iPSC therapy candidate to be licensed from the strategic research and development alliance that FUJIFILM Cellular Dynamics, Opsis Therapeutics, and BlueRock Therapeutics entered in 2021 to discover and develop off-the-shelf iPSC-derived cell therapies for ocular diseases. The strategic alliance grants BlueRock Therapeutics the exclusive option to license three retinal cell therapy programs focused on dry age-related macular degeneration (AMD) and inherited retinal diseases (IRD).

A leading cause of permanent vision impairment and blindness worldwide is the degeneration and loss of photoreceptor cells (rods and cones) in the retina due to age, injury, or IRD. As these photoreceptors are not able to regenerate in humans, one solution is to replace them with a cellular therapy. OpCT-001 is composed of allogeneic human iPSC-derived photoreceptor cells, that once transplanted, are intended to aid in recovery of vision by maturing into cones (responsible for central high acuity and color vision), and rods (responsible for vision at low light levels and peripheral vision).

“We have collaborated closely with BlueRock Therapeutics to develop a new generation of iPSC-derived cell therapies and we are pleased to grant this license as the next step towards moving the OpCT-001 product into the clinic for the treatment of human retinal diseases” said Tomoyuki Hasegawa, president and chief executive officer, of FUJIFILM Cellular Dynamics. “We continue to have a productive partnership with BlueRock Therapeutics and look forward to unlocking the full potential of iPSC technology to create best-in-class cell replacement therapies targeting ocular diseases.”

“The development of iPSC-derived therapies is an exciting scientific frontier that has great potential for restoring vision in patients living with vision impairment and blindness,” said Seth Ettenberg, president and chief executive officer of BlueRock Therapeutics. “This agreement is an exciting step in the maturation of our collaboration with the FUJIFILM Cellular Dynamics and Opsis Therapeutics team, and we are thrilled to be advancing OpCT-001 to the clinic, with an IND filing planned for later this year.”

FUJIFILM Cellular Dynamics is committed to supporting the cell therapy market, and recently announced an investment that doubles its existing manufacturing capacity for the development and commercialization of iPSC-derived cell therapies. The new site is expected to be fully operational in 2026. To learn more please refer to the press release: https://www.fujifilmcdi.com/news-item/fujifilm-to-invest-200-million-to-expand-cell-therapy-development/

AMD is the leading cause of irreversible blindness and visual impairment in the world, and experts predict that the number of people affected worldwide will reach 288 million by 2040^*1. IRDs are the leading cause of vision loss in persons between 15 and 45 years of age^*2.

FUJIFILM Cellular Dynamics, Inc. is a leading developer and manufacturer of human iPSCs and iPSC-derived cells utilized in drug discovery, contract development and manufacturing services for cell therapies. For its partners, FUJIFILM Cellular Dynamics utilizes its iPSC platform to advance the progress of therapeutic candidates in the clinic and provides contract development and manufacturing (CDMO) services. In addition to cell therapy, FUJIFILM Cellular Dynamics also offers life science research tools including the company’s inventoried iCell® products, which are available in almost any cell type and are sourced from multiple cell lines which can be applied for target identification as well as toxicity testing. The company also offers custom cell services and cell banking. FUJIFILM Cellular Dynamics’ goal is to leverage the vast utility of iPSCs to advance human health and improve the quality of life for patients around the world. For more information, please visit: https://www.fujifilmcdi.com/

Opsis Therapeutics is a company focused on advancing the pipeline of cell replacement therapies targeting ocular diseases. Leveraging innovations in induced iPSC generation, retinal cell differentiation, and functional biomaterials, Opsis Therapeutics is developing cell therapies for patients with dry AMD, inherited macular degenerations (IMDs) and IRDs. Founded in 2016, Opsis Therapeutics is a joint venture of FUJIFILM Cellular Dynamics and David Gamm, M.D., Ph.D., and is headquartered in Madison, Wisconsin. For more information, please visit: www.opsistx.com.

FUJIFILM Holdings Corporation, Tokyo, leverages its depth of knowledge and proprietary core technologies to deliver “Value from Innovation” in our products and services in the business segments of healthcare, materials, business innovation, and imaging. Our relentless pursuit of innovation is focused on providing social value and enhancing the lives of people worldwide. Fujifilm is committed to responsible environmental stewardship and good corporate citizenship. For more information about Fujifilm’s Sustainable Value Plan 2030, click here. For the year ended March 31, 2023, the company had global revenues of approximately 2.9 trillion yen (21 billion USD at an exchange rate of 134 yen/dollar). For more information, please visit: https://holdings.fujifilm.com/en.

• *1 “Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis.” Wong, Wan Ling, et al. Wong, Wan Ling, et al. The Lancet Global Health, vol. 2, no. 2, 2014, doi:10.1016/s2214-109x(13)70145-1.
• *2 Genes (Basel). 2018 Apr; 9(4): 215. Published online 2018 Apr 14. doi: 10.3390/genes9040215

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