ReviR Therapeutics Secures Investment from CMT Research Foundation to Advance First-in-Class Oral Small Molecule RNA-Targeted Therapy for CMT1A
ReviR Therapeutics
Innovative Drug Developer
On June 19, ReviR Therapeutics (hereinafter referred to as "ReviR") and a non-profit organization dedicated to advancing research on therapies for Charcot-Marie-Tooth disease (CMT) and patient treatmentCMT Research Foundation (CMTRF)Announced the establishment of a strategic partnership,ReviR Therapeutics Receives Funding Support from CMTRF, will be in cooperationAdvancing the Research of Small Molecule Targeted RNA Therapy for Regulating CMT1A Pathogenic Gene Expression。
ReviR TherapeuticsOral Gene TherapyWith the goal of research and development, and leveraging the proprietary AI drug discovery platform VoyageR, the company is currently focused on developing a series of small-molecule drugs that modulate splicing—SpliceR. These drugs regulate gene expression by impacting mRNA transcripts to achieve therapeutic effects.In the collaboration project with CMTRF, ReviR will develop splice modulators to reduceCMT1AOverexpression of pathogenic genes, thereby alleviating disease symptoms.Compared with other therapies that require injection, this drug has the advantage of being orally administrable, making it more convenient for delivery.This innovative therapy is not only set to revolutionize the treatment paradigm for CMT1A but also holds promise for application in other subtypes of CMT and other neurodegenerative diseases.
ReviR CEO and co-founder Dr. Peng Yue stated, "ReviR's technology and therapeutic approach are fundamentally different from traditional high-invasiveness gene therapies.This is an orally available drug that can cross the blood-brain barrier and directly interact with mRNA.Thereby regulating splicing. Changes in splicing can disrupt mRNA, effectively reducing the expression of pathogenic gene proteins.Block the occurrence of CMT1A at the genetic level.”
CMTRF CEO Cleary Simpson stated, "CMTRF has always been committed to accelerating the development of therapies that can halt or even reverse the progression of CMT. Our collaboration with ReviR perfectly aligns with our mission. Their cutting-edge technology holds promise for providing aHighly efficient and easy to administerThe innovative therapy may bring new improvements to the lives of those suffering from this chronic disease."
This project will validate the activity of splicing agents in CMT1A cell models through experiments, rapidly advancing the pipeline to animal testing and ultimately proceeding to clinical trials.
"I have dedicated the past decade to researching new ways to safely and effectively treat patients with CMT, and for this research project, we will move forward with determination," said Dr. Paul August, Chief Scientific Officer (CSO) of ReviR Therapeutics and member of the CMTRF Scientific Advisory Board (SAB). "Our goal is not only innovation but also applying that innovation to treatment methods that can bring tangible benefits to patients. By studying oral medications that reduce the expression of disease-causing proteins, we hope to provide a treatment that is both effective and suitable for daily life, minimizing the psychological and physical burdens caused by CMT1A as much as possible."
He further explained, "The drug developed by ReviR, called SpliceR, is highly groundbreaking and belongs toNovel Gene Therapy. This therapyAffecting Proteins by Regulating Gene Expression, which is similar to the therapeutic mechanism of Risdiplam, a drug approved by the FDA. Risdiplam achieves its therapeutic purpose by modulating gene expression to produce an important protein needed by the body, while ReviR's splicing agents also regulate gene expression, but in a different manner.Splice-switching agents halt the production of disease-causing proteins by inserting a genetic signal that prompts cells to stop expressing the protein at the right time.This approach, compared to other gene therapies, is able toThe treatment burden is lighter and accessibility is higher.”
(From left to right) Dr. Paul August, Cleary Simpson, Dr. Peng Yue
*Photographed at the 2024 BIO International Convention
About CMTRF
The Charcot-Marie-Tooth Research Foundation (CMTRF) is a patient-led nonprofit organization focused on advancing therapy research for CMT and achieving treatments for CMT. In response to the difficulties and obstacles encountered during the process of CMT therapy research, the foundation actively seeks collaborators to resolve them. To date, CMTRF has funded 24 projects, eight of which have been successfully completed. Among the completed projects, five have developed clinical candidate drugs, one of which, DTx Pharma's candidate drug, was acquired by Novartis for $1 billion. The foundation was established by two patients who are eager to accelerate CMT treatment and is supported by donations from individuals and enterprises.
*Charcot-Marie-Tooth disease (CMT) is a group of hereditary peripheral neuropathies that cause nerve degeneration. The leg and arm muscles of patients gradually atrophy, leading to abnormal hand and foot function, limited balance ability, and difficulties in walking, running, etc. The prevalence of CMT is approximately 1/2500, affecting nearly 3 million people worldwide, with about 150,000 cases in the United States. Currently, there are no treatments or cures available for CMT.
