The CMT Research Foundation, committed to finding treatments for Charcot-Marie-Tooth disease (CMT), has teamed up with ReviR Therapeutics. Their collaboration aims to develop small molecule therapies that can adjust the expression of the gene linked to CMT1A.

ReviR Therapeutics is at the forefront of developing small molecule splice modulators, which can control which parts of the genetic code are translated into proteins. Supported by CMTRF, this initiative focuses on creating a splice modulator to lower the expression of the gene responsible for CMT1A, addressing the symptoms of the disease. A key benefit of this treatment is its oral form, making it easier for patients compared to injectable alternatives. The innovative technologies from ReviR Therapeutics could revolutionize treatments not only for CMT1A but also for other types of CMT and neurodegenerative disorders.

"Our platform technology represents a significant departure from traditional genetic therapies that often require invasive delivery methods," says Peng Yue, Ph.D., CEO and Co-founder of ReviR Therapeutics. "We've developed small molecules that can be taken by mouth, cross the blood-nerve barrier, and directly interact with mRNA to alter splicing. This modification results in mRNA degradation, effectively reducing the production of disease-causing proteins."

"Partnering with ReviR Therapeutics is in perfect harmony with CMTRF's goal of accelerating the development of therapies that could stop or reverse the progression of CMT," states Cleary Simpson, CEO of CMTRF. "Their cutting-edge technology has the potential to dramatically improve the lives of those affected by this chronic condition by offering a non-invasive, effective, and convenient treatment option."

The project will first test the effectiveness of these splicing modulators in cell models of CMT1A, with plans to advance to animal studies and eventually clinical trials.

"We are deeply committed to this research, building on a decade of work exploring new ways to treat CMT patients safely and effectively," adds Dr. Paul August, Chief Scientific Officer of ReviR Therapeutics and a member of CMTRF's scientific advisory board. "Our objective is to not only innovate but also to apply our innovations in ways that provide tangible benefits to patients. By reducing disease-causing protein levels with an orally administered drug, we aim to deliver a therapy that is both effective and fits into patients' daily lives, thus reducing the emotional and physical burden of CMT1A."

"ReviR Therapeutics has developed a pioneering category of medication, SpliceR, which aligns with other advanced genetic treatments," explains Dr. August. "Similar to the FDA-approved drug Risdiplam for spinal muscular atrophy, our SpliceR drugs modify the genetic instructions by adding a signal to stop protein production, thereby reducing harmful proteins. This approach could be a significant advancement over existing gene therapies, making treatment less burdensome and more accessible."

 

Source: prnewswire.com

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