ReviR Therapeutics Receives Investment from CMT Research Foundation, Pioneering Small-Molecule RNA-Targeted Therapy for CMT1A | BlueRun Ventures Headlines

On June 19, ReviR Therapeutics ("ReviR") and the CMT Research Foundation (CMTRF), a nonprofit organization dedicated to advancing therapies and patient care for Charcot-Marie-Tooth disease (CMT), announced a strategic partnership to jointly advance research into small-molecule RNA-targeted therapies that modulate expression of the CMT1A disease-causing gene.

ReviR is developing a pipeline of small-molecule drugs that regulate splicing — called SpliceRs — which modulate gene expression by influencing mRNA transcripts to achieve therapeutic effects. With CMTRF's support, ReviR will develop SpliceRs to reduce overexpression of the CMT1A disease-causing gene, thereby alleviating disease symptoms. Compared to other therapies requiring injection, this drug offers the advantage of oral administration, enabling more convenient delivery. This innovative therapy will not only transform the treatment paradigm for CMT1A but also holds potential for application in other CMT subtypes and other neurodegenerative diseases.

Dr. Peng Yue, CEO and Co-founder of ReviR, stated: "ReviR's technology and therapeutic approach are fundamentally different from traditional highly invasive gene therapies. This is an orally available drug capable of crossing the blood-brain barrier to directly interact with mRNA and modulate splicing. Changes in splicing can destroy mRNA, effectively reducing expression of the disease-causing protein and blocking CMT1A at the genetic level."

Cleary Simpson, CEO of CMTRF, stated: "CMTRF has always been mission-driven to accelerate the development of therapies that can halt or even reverse CMT disease progression. Our partnership with ReviR aligns perfectly with that mission. Their cutting-edge technology promises to deliver an innovative therapy that is both effective and easy to administer, potentially bringing meaningful improvements to the lives of those living with this chronic condition."

The project will validate SpliceR activity in CMT1A cellular models in experiments, with the goal of rapidly advancing the pipeline to animal studies and ultimately clinical trials.

"I have spent the past decade working to find new ways to safely and effectively treat CMT patients, and we are committed to advancing this research program," said Dr. Paul August, Chief Scientific Officer of ReviR and member of CMTRF's Scientific Advisory Board (SAB). "Our goal is not just innovation, but translating that innovation into treatments that deliver tangible benefits to patients. By researching an oral drug that reduces expression of the disease-causing protein, we hope to provide a therapy that is both effective and compatible with daily life, minimizing the psychological and physical burden of CMT1A as much as possible."

He further explained: "The drug ReviR is developing, called SpliceR, is highly groundbreaking and represents a novel form of gene therapy. This therapy influences proteins by modulating gene expression, with a mechanism of action similar to the FDA-approved drug Risdiplam. Risdiplam achieves its therapeutic effect by regulating gene expression to produce an essential protein the body needs; ReviR's SpliceR similarly modulates gene expression, but through a different approach. The SpliceR inserts a genetic signal that instructs the cell to stop expressing the protein at the appropriate time, thereby preventing production of the disease-causing protein. Compared to other gene therapies, this approach offers a lighter treatment burden and greater accessibility."

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