CMT Research Foundation Funds ReviR Therapeutics for CMT1A Small Molecule Treatment

The CMT Research Foundation (CMTRF), based in Atlanta, is a non-profit organization dedicated to finding treatments and cures for Charcot-Marie-Tooth disease (CMT). In a significant development, CMTRF has announced a strategic partnership with ReviR Therapeutics aimed at advancing small molecule therapeutics to modify the expression of the gene responsible for CMT1A, a common form of CMT.

ReviR Therapeutics specializes in developing small molecule splice modulators. These drugs work by altering how genetic information is processed within cells, specifically affecting which parts of the genetic code are included in the final RNA transcript. The goal of this partnership is to create a splice modulator that reduces the expression of the overactive gene causing CMT1A symptoms. One of the key advantages of this approach is that these drugs can be taken orally, providing a more convenient alternative to injection-based treatments.

ReviR Therapeutics' approach holds the potential to transform the treatment landscape not just for CMT1A, but also for other types of CMT and neurodegenerative diseases. Peng Yue, Ph.D., CEO and Co-founder of ReviR Therapeutics, emphasized that their platform technology represents a significant departure from traditional genetic therapies, which often require invasive administration methods. He highlighted that their small molecules are designed to be taken orally, cross the blood-nerve barrier, and directly interact with mRNA to modulate splicing. This interaction leads to the destruction of mRNA and a subsequent reduction in disease-causing protein levels.

Cleary Simpson, CEO of CMTRF, expressed enthusiasm about the collaboration, noting that it aligns perfectly with CMTRF's mission to fast-track the development of therapies that could halt or even reverse the progression of CMT. Simpson highlighted the potential of ReviR's technology to provide a non-invasive, potent, and easily administrable treatment option for those suffering from this chronic condition.

The partnership will involve experiments to validate the effectiveness of these splicing modulators in cell models of CMT1A, with plans to quickly advance to animal studies and eventually clinical trials. Dr. Paul August, Chief Scientific Officer of ReviR Therapeutics and a member of CMTRF's scientific advisory board, stressed the importance of this research program. He noted that their aim is not just to innovate but to deliver real, tangible benefits to patients by reducing disease-gene protein levels through an orally administered drug, thereby minimizing the psychological and physical toll of CMT1A.

ReviR Therapeutics has developed a novel type of medication known as SpliceR, which falls under a new category of genetic treatments. These treatments modify the genetic information used to produce proteins, similar to how the FDA-approved drug Risdiplam treats spinal muscular atrophy. However, SpliceR drugs add a stop signal to the genetic message, leading to the reduction of problematic proteins. This innovative approach could represent a significant improvement over existing gene therapies, making treatment less burdensome and more accessible.

Founded in 2021 and headquartered in Brisbane, CA, ReviR Therapeutics is a biopharmaceutical company focused on RNA-targeting small molecule therapies for CNS diseases, cancer, and other rare genetic conditions. The company's initial focus is on SpliceR modulation technologies aimed at genetic diseases with limited or no treatment options.

The CMTRF is a patient-led non-profit organization committed to delivering treatments and cures for CMT. The foundation seeks to identify and address significant obstacles in the path to a cure. To date, CMTRF has funded 24 projects, with eight completed and five clinical candidates emerging from those projects. The foundation's mission is supported by personal and corporate financial contributions.

Charcot-Marie-Tooth disease is a group of inherited, chronic peripheral neuropathies leading to nerve degradation. CMT patients experience progressive muscle atrophy in their legs and arms, resulting in mobility and balance issues, as well as abnormal functioning of hands and feet. Affecting about one in 2,500 people, including 150,000 Americans and nearly 3 million globally, CMT currently has no cure or treatment.