ReviR Therapeutics Secures $4.6M CIRM Grant to Advance HTT-PMS1 Dual-Target RNA-Directed Small Molecule Program for Huntington’s Disease
ReviR Therapeutics
Innovative Drug Developer
California Institute for Regenerative Medicine
The California Institute for Regenerative Medicine was established in early 2005 following the passage of Proposition 71 (the California Stem Cell Research and Cures Initiative). On November 2, 2004, California voters approved a statewide ballot measure that provided $3 billion in funding for stem cell research at the University of California and other research institutions, and called for the creation of a new state agency to provide grants and loans for stem cell research, research facilities, and other critical research opportunities. CIRM’s mission is to support and advance stem cell research and regenerative medicine under the highest ethical and medical standards, with the aim of discovering and developing treatments, therapies, diagnostics, and research technologies to alleviate human suffering caused by chronic diseases and injuries.
November 20, 2025ReviR Therapeutics (ReviR Therapeutics, Inc., referred to as ReviR) announced that it has received a $4.6 million grant from CIRM (California Institute for Regenerative Medicine)., for the advancement of the company's independently developed "HTT-PMS1" innovative project research. The project isSmall-molecule targeted RNA dual-target pipeline, indicated for Huntington's disease(Huntington's Disease, HD), has recently successfully obtained a lead compound that meets the standards.Achieve the First Core Milestone。
Huntington's disease is a neurodegenerative disorder caused by the abnormal expansion of CAG trinucleotide repeats in the HTT gene. Typically, patients have more than 36 CAG repeats in their HTT gene, leading to the formation of an abnormal polyglutamine (PolyQ) chain that produces mutant huntingtin protein (mHTT). This abnormal protein misfolds and aggregates within neurons, disrupting various cellular functions and causing neuronal damage. Recent studies indicate that although the length of CAG repeats is determined at birth, these sequences can still expand in certain brain cells. Once the expansion exceeds a critical threshold (approximately 150 CAG repeats), it triggers neuronal death and accelerates disease progression.
Mutant huntingtin protein (mHTT) and mismatch repair enzyme PMS1 are two key drivers in the pathogenesis of Huntington's disease.ReviR Therapeutics leverages its self-developed VoyageR AI platform and cutting-edge RNA-targeting technology to simultaneously target these two key factors. By using small molecules to induce RNA degradation, it reduces toxic protein levels and inhibits CAG repeat expansion, potentially blocking the long-term progression of the disease at its root.
CIRM is one of the most important public funding agencies for regenerative medicine globally, awarding grants through a rigorous peer-review process.This recognition fully reflects ReviR's scientific research strength, innovative potential, and clinical transformation value in the field of neurodegenerative diseases.This funding will be used for preclinical research and clinical trials of ReviR's candidate drugs, accelerating the development of innovative therapies that can effectively slow disease progression and are convenient for patients to use, bringing a new treatment paradigm for neurodegenerative diseases.
ReviR Therapeutics Chief Scientific Officer (CSO) Paul R. August, Ph.D., stated: "The CIRM grant provides crucial support for our development of targeted therapies for Huntington's disease. Leveraging proprietary RNA-targeting technology and the VoyageR AI platform,The small molecule drugs we have developed are expected to revolutionize the treatment of Huntington's disease."，This oral therapy, once a breakthrough is achieved, will significantly improve patient compliance and quality of life, bringing hope for a fundamental cure."
Dr. Peng Yue, co-founder and CEO of ReviR, stated: "We sincerely thank CIRM for their recognition and funding. This funding will advance our 'HTT-PMS1' dual-target project into the clinical stage. I believeThis will not only change the fate of Huntington's disease patients but also benefit more patients with other neurodegenerative diseases.”
About ReviR Therapeutics
ReviR Therapeutics is a global biotechnology company dedicated to developing innovative therapies for patients with neurogenetic diseases worldwide. Established in 2021, the company’s core team members have deep expertise in computational biology, AI, RNA biology, and drug discovery. ReviR leverages its cutting-edge technology and AI-driven drug discovery platform, VoyageR, to develop highly specific, effective, and safe disease-modifying therapies. Its lead small-molecule candidate, RTX-117, is currently advancing in clinical trials for Charcot-Marie-Tooth disease (CMT) while expanding into new indications. The pipeline also includes multiple other small-molecule drugs that achieve therapeutic effects by modulating RNA function.
Official Website: www.revirtx.com
Contact for inquiries: info@revirtx.com
Media/Partnership Contact: pr@revirtx.com
About CIRM
CIRM (California Institute for Regenerative Medicine) is one of the largest public funding institutions globally, aiming to accelerate the delivery of therapies to patients with unmet medical needs by supporting stem cell and gene therapy research. Over the past 20 years since its establishment, CIRM has played a significant role in advancing the development of breakthrough therapies, bringing substantial positive impacts to global health. For more information, please visit www.cirm.ca.gov.