Advancement of novel RNA Therapeutic will leverage Enzymatic Synthesis Platform from EnPlusOne.
EnPlusOne Biosciences is to be part of a collaboration led by the Wyss Institute for Biologically Inspired Engineering at Harvard University that has been awarded an agreement for up to $27 million by the Advanced Research Projects Agency for Health (ARPA-H).
The agreement covers multi-disciplinary efforts to develop a disease-agnostic novel RNA therapeutic with the potential to treat diverse diseases, including types of cancer and infectious diseases – and to be effectively and rapidly deployable.
Initially, up to $3.5 million of the agreement is focused on the EnPlusOne enzymatic platform.
ARPA-H is a federal funding agency which funds transformative biomedical and health research breakthroughs, translating prioritized research from the lab to applications in the marketplace.
The ARPA-H award will allow the Wyss Institute team to significantly accelerate and expand efforts to advance the therapy towards an Investigational New Drug (IND) submission to the Federal Food and Drug Administration (FDA).
The team is initially focusing on cancer and will explore the platform’s potential for difficult-to-treat infectious diseases.
The ARPA-H project will build on a newly developed Duplex RNA technology and leverage the powerful RNA delivery capabilities developed by groups at the Wyss Institute, along with the groundbreaking enzymatic RNA synthesis capabilities of EnPlusOne to optimize the RNA’s design and scalability.
Dan Ahlstedt, co-founder and Chief Operating Officer, EnPlusOne Biosciences, said: “We come full circle rejoining our Wyss colleagues for this exciting collaboration. This comprehensive program will give us the opportunity to demonstrate how enabling modifications and limitless scale can be unlocked by our ezRNA platform. We are grateful to the Wyss
Institute and ARPA-H for recognizing the current problems facing RNA manufacturing and sharing our vision that an enzymatic approach is the future. We look forward to progressing our platform alongside this team as we collectively work to address critical human health challenges.”
Wyss Institute Core Faculty member Natalie Artzi, Ph.D. is the lead-investigator on the project with co-principal investigator and Wyss Institute Founding Director Don Ingber, M.D., Ph.D.
Artzi also is Associate Professor of Medicine at Harvard Medical School (HMS) and Brigham and Women’s Hospital and a Principal Research Scientist at MIT.
Additional key investigators include Wyss Institute Director of Translational R&D Kenneth Carlson, Ph.D., a drug discovery and development specialist with extensive industry experience and Wyss Institute Core Faculty member William Shih, Ph.D., who developed a DNA origami platform that allows the precise and highly effective presentation of RNA drugs, cancer and pathogen-derived antigens and immune activating adjuvants to the immune system.
Shih’s team will provide their DNA nanotechnology approach as an additional drug delivery option.
The collaboration is another validation of the potential for enzymatic synthesis to address the needs of a rapidly expanding RNA therapeutic market as the industry faces demand for RNA production that cannot be met by chemical synthesis alone. It builds upon recent important progress shared by EnPlusOne, including the breakthrough synthesis of the antisense strand of the commercially approved siRNA drug, Leqvio (inclisiran), a treatment for hypercholesteremia (high cholesterol) licensed from Alnylam Pharmaceuticals, Inc. by Novartis, that currently addresses a multi-million patient population.
EnPlusOne was launched by the Wyss Institute in 2022 to commercialize its enzymatic RNA oligonucleotide synthesis technology developed in the laboratory of co-founder George Church, PhD.
Seed financing was led by Northpond Ventures, with participation from Breakout Ventures, Coatue and individual investors.