Risdiplam

ATLANTA, June 19, 2024 /PRNewswire/ -- The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has formed a strategic partnership with ReviR Therapeutics to advance small molecule therapeutics that modulate the expression of the gene that causes CMT1A. ReviR Therapeutics is developing a class of drugs called small molecule splice modulators, which can regulate the expression of a gene by influencing which components of the genetic code get included in the final version of the RNA transcript. In this project supported by the CMTRF, ReviR Therapeutics will develop a splice modulator that will reduce the expression of the disease gene, which is overexpressed in CMT1A and is the cause of disease symptoms. A benefit of this type of drug is that it can be taken orally, making it much more convenient for patients compared to the injections required for other approaches. The technologies and approaches from ReviR Therapeutics could revolutionize treatment paradigms not only for CMT1A but potentially for other forms of CMT and other neurodegenerative disorders as well. "Our platform technology at ReviR represents a radical departure from traditional genetic therapies that often require invasive administration methods," says Peng Yue, Ph.D., Chief Executive Officer and Co-founder of ReviR Therapeutics. "We have designed small molecules that can be taken orally, cross the blood-nerve barrier, and directly interact with mRNA to modulate splicing. The splicing change will cause mRNA destruction and will effectively reduce disease-gene protein levels to address the genetic basis of CMT1A." "Collaborating with ReviR Therapeutics aligns perfectly with CMTRF's mission to accelerate the development of therapies that can halt or even reverse the progression of CMT," says Cleary Simpson, CEO of the CMTRF. "Their cutting-edge technology has the potential to transform the lives of those living with this chronic condition by providing a non-invasive, potent, and easily administrable treatment option." The experiments in this project will validate the effectiveness of these splicing modulators in cell models of CMT1A, with the goal to quickly advance to animal studies and, eventually, clinical trials. "We are deeply committed to this research program and I've spent the last decade examining new approaches to treat CMT patients safely and effectively," Dr. Paul August, Chief Scientific Officer of ReviR Therapeutics and who serves on CMTRF's scientific advisory board, adds, "Our goal is not just to innovate but to apply our innovations in ways that offer real, tangible benefits to patients. By reducing disease-gene protein levels with an orally administered drug, we hope to provide a therapy that is both effective and aligns with everyday life, minimizing the psychological and physical toll of CMT1A." "ReviR Therapeutics has developed a groundbreaking type of medication called SpliceR, which belongs to a new category of genetic treatments. These treatments work by tweaking the process of how genetic information is used to make proteins, a process somewhat similar to the way the FDA-approved drug Risdiplam treats spinal muscular atrophy. Risdiplam helps by adjusting a gene to produce a vital protein needed by the body," explains Dr. August. "Similarly, ReviR's SpliceR drugs make adjustments to the genetic information but in a different way. SpliceR drugs add a piece to the genetic message that tells the body to stop expressing the protein and the position of this stop signal leads to the reduction of problematic proteins. This approach could be a big step forward compared to some other gene therapies, making treatment less burdensome and more accessible." ReviR Therapeutics is an emerging biopharmaceutical company focused on the development of RNA-targeting small molecule therapies that modulate the causal genes of CNS diseases, cancer, and other rare genetically defined diseases. The company is utilizing cutting-edge technologies to develop therapies with the goal of developing disease modifying therapies that are efficacious, safe, and specific. The company's initial focus is to apply SpliceR modulation technologies towards RNA targets of genetic diseases with limited or no treatment options. ReviR Therapeutics was founded in 2021 and headquartered in Brisbane, CA. CMT Research Foundation (CMTRF) is a patient-led, non-profit focused on delivering treatments and cures for CMT. The foundation identifies significant obstacles or deficiencies impeding progress toward a cure and seeks out collaborators to address these issues. To date, CMTRF has funded 24 projects, of which 8 are completed. Of those 8 completed projects, 5 have clinical candidates. CMTRF's mission to invest in promising science with high potential of leading to treatments and cures was proven effective and ground-breaking when DTx Pharma with a CMTRF- backed program as its lead candidate was acquired by Novartis for $1 billion. Founded by two patients who are driven to expedite drug delivery to people who live with CMT, the 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts. * Charcot-Marie-Tooth encompasses a group of inherited, chronic peripheral neuropathies that result in nerve degradation. CMT patients suffer from progressive muscle atrophy of legs and arms, causing walking, running and balance problems as well as abnormal functioning of hands and feet. CMT affects one in 2,500 people (about the same prevalence as cystic fibrosis), including 150,000 Americans and nearly 3 million people worldwide. At the moment, there is no treatment or cure for CMT. SOURCE ReviR Therapeutics