February 14, 2023, San Diego, California — Ryne Biotechnology, Inc. (Ryne Bio), a therapeutics company leveraging induced pluripotent stem cell (iPSC) technology to discover and develop a platform of off-the-shelf neuron replacement therapies for neurological disorders, announced that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4 million Clinical Stage Research Program (CLIN1) grant. This funding will enable the company to advance its lead candidate RNDP-001, an iPSC-derived dopamine neuron progenitor for the treatment of both inherited and idiopathic forms of Parkinson’s disease, through submission of an Investigational New Drug (IND) application within the next 12 months.
RNDP-001 has completed preclinical efficacy and safety studies. This CIRM award will allow Ryne Bio to finalize its IND-package including the production of GMP-grade materials to enable the evaluation of RNDP-001 in Phase 1 clinical trials for both inherited and idiopathic forms of Parkinson’s disease.
“We appreciate CIRM’s partnership in our vision to reverse degenerative conditions of the brain by developing off-the-shelf cell replacement therapies,” said Nick Manusos, Chief Executive Officer of Ryne Bio. “A dramatic shift in the standard of care for patients with neurodegenerative disease is long overdue. We are thrilled to be developing groundbreaking therapies for patients in need of better treatment options.”
Beyond RNDP-001, Ryne Bio is developing a platform of drug candidates, including next-generation, gene-modified programs that have the potential to modify and reverse disease progression in Parkinson’s disease and other moderate to severe central nervous system disorders.
“The underlying cause of Parkinson’s disease is progressive degeneration of a patient’s dopamine neurons. Ryne Bio is able to directly replace dopamine neurons that have been lost by utilizing precision manufacturing techniques,” said Howard Federoff, M.D., Ph.D., Ryne Bio’s Chief Medical Officer, scientific co-founder and Principal Investigator on the CLIN1 award.
In addition to funding from CIRM, Ryne Bio was launched and seeded in 2022 by Saisei Ventures, an emerging venture capital firm focused on building revolutionary advanced medicine companies. “The potential of off-the-shelf cell replacement therapies is on the cusp of being realized for complex and intractable disease,” said Jonathan Yeh, Managing Partner of Saisei Ventures. “This funding decision from CIRM provides robust validation of the Ryne Bio approach, and supports the delivery of this best-in-class therapy to patients.”
About Ryne Bio
Ryne Bio is a biotechnology company pioneering next-generation approaches to cure neurological disorders. The company utilizes induced pluripotent stem cell (iPSC) technology, a Nobel-winning breakthrough that enables scientists to manufacture any human cell, in order to advance Ryne Bio’s off-the-shelf neuron replacement therapeutics. By focusing on an iPSC technology platform, and forging partnerships with global leaders in surgical delivery and clinical development, Ryne Bio is dedicated to advancing a best-in-class pipeline targeting neurological diseases.
About the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality.
About Saisei Ventures
Saisei Ventures is a leading venture capital firm dedicated to building next-generation companies in the healthcare sector. It aims to partner with passionate bio-entrepreneurs to develop and implement business strategies that will generate strong proof of concept, clinical validation, and market value. With operations in Japan and the United States, Saisei aims to enhance the value of its portfolio companies by leveraging its unique networks in and the institutional advantages of both countries. Saisei’s first fund, Saisei Bioventures L.P., is focused on building revolutionary cell and gene therapy and regenerative medicine companies from foundational technologies originating from Japan.
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