ReviR Therapeutics Secures Kennedy’s Disease Association Grant to Advance Therapy for Rare Neuromuscular Disorder

26 November 2024 | Tuesday | News


Collaboration Targets Mutant mRNA Splicing to Develop Transformative Treatment for Spinal and Bulbar Muscular Atrophy (Kennedy's Disease)
Image Source : Public Domain

Image Source : Public Domain

ReviR Therapeutics, a pioneering biotechnology company focused on developing novel small molecule RNA modulators for neurogenetic diseases, announced a groundbreaking research grant from the Kennedy's Disease Association (KDA) to advance a therapy for spinal and bulbar muscular atrophy (SBMA), a rare neuromuscular disorder currently lacking effective treatments. SBMA is also known as Kennedy's Disease (KD) named after Dr. William R. Kennedy, who first described the condition.

The collaboration aims to identify a small molecule that targets mutant androgen receptor (AR) mRNA splicing and causes selective destruction of the disease-causing mRNA. By modulating mRNA splicing, ReviR aims to develop a potential therapeutic intervention for Kennedy's Disease (KD).

"Our proprietary VoyageR platform enables us to identify molecules that induce the destruction of disease mRNA by incorporating a novel pseudo exon, effectively triggering nonsense-mediated decay of the respective mRNA in a manner similar to oligonucleotide therapeutics," said Paul R. August, PhD, Chief Scientific Officer at ReviR Therapeutics. "This breakthrough approach has the potential to significantly reduce toxic disease protein levels and modify disease progression for KD patients."

"We are excited to partner with the Kennedy's Disease Association to advance our research efforts and bring hope to the KD community," said Peng Yue, PhD, CEO at ReviR Therapeutics. "By combining our scientific expertise with the KDA's deep understanding of patient needs, we are confident in our ability to develop a transformative therapy for KD."

"KDA is excited to partner with ReviR Therapeutics on the potential use of its innovative gene editing technology as a novel therapeutic strategy for Kennedy's Disease. Reducing or eliminating the toxic protein that causes the disease will be life-changing for people living with KD. We look forward to a successful outcome and a long-term relationship with ReviR," said KDA President Terry Thompson, PhD. ReviR's commitment to scientific excellence and patient-centric approach, coupled with the KDA's dedication to supporting individuals with KD, positions this partnership to make a substantial impact on the field of neurodegenerative diseases. The outcomes of this research will not only advance the understanding of disease mRNA regulation but also accelerate the development of innovative therapies for KD

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