ReviR Therapeutics, an innovative biotech firm specializing in creating unique small molecule RNA modulators to combat neurogenetic disorders, has received a pivotal research grant from the Kennedy’s Disease Association (KDA). This collaboration focuses on pioneering a treatment for spinal and bulbar muscular atrophy (SBMA), also known as Kennedy’s disease, a rare neuromuscular condition that currently lacks effective therapies. Named after Dr. William R. Kennedy, who first identified the disorder, this initiative seeks to revolutionize treatment options.
The partnership’s prime objective is to discover a small molecule capable of targeting the mutant androgen receptor (AR) mRNA splicing, leading to the selective elimination of the harmful mRNA. ReviR is hopeful that by adjusting the mRNA splicing process, they can create a viable treatment for Kennedy’s disease.
Paul R. August, PhD, chief scientific officer at ReviR Therapeutics, explained, “Our innovative VoyageR platform allows us to pinpoint molecules that instigate the breakdown of disease-specific mRNA. This is achieved by integrating a novel pseudo exon, initiating a nonsense-mediated decay, akin to oligonucleotide therapies. This transformative technique holds promise in dramatically lowering levels of the toxic proteins linked to the disease, potentially altering its progression in KD patients.”
“We are thrilled to join with the Kennedy’s Disease Association in pushing our research further, bringing optimism to those impacted by KD,” stated Peng Yue, PhD, CEO at ReviR Therapeutics. “By merging our advanced scientific knowledge with the KDA’s thorough patient insights, we’re dedicated to developing an impactful treatment for this condition.”
“KDA is eager to collaborate with ReviR Therapeutics in exploring its cutting-edge gene-editing technology as a fresh approach to treating Kennedy’s disease. Eradicating or minimizing the toxic protein responsible for the illness could profoundly impact the lives of those with KD. We anticipate both a successful project and a lasting alliance with ReviR,” shared Dr. Terry Thompson, President of the KDA. The combination of ReviR’s commitment to scientific excellence and patient-focused research, alongside the KDA’s ongoing mission to support those with KD, positions this partnership to bring about meaningful advancements in neurodegenerative disease treatment.
ReviR Therapeutics is a rising entity in the biopharmaceutical landscape, focusing on devising RNA-targeted small molecules that impact the genetic roots of diseases, including central nervous system ailments, cancer, and other rare genetic conditions.
The Kennedy’s Disease Association, a non-profit organization, is devoted to bettering the lives of individuals with Kennedy’s Disease. It offers support, education, and advocacy for patients and their families while funding research to quicken the development of effective KD treatments.