Revir wins $4.6M grant to advance oral Huntington’s treatment
Company says funding will help advance 'life-changing therapy'
Revir Therapeutics has been awarded a $4.6 million grant from the California Institute for Regenerative Medicine (CIRM) to support the development of a potential disease-modifying oral treatment for Huntington’s disease.
The company also said it “reached a key program milestone,” identifying “a lead compound that meets key development criteria.”
Revir, through its HTT-PMS1 program, aims to develop a small molecule that selectively reduces the production of two proteins implicated in both the cause and progression of Huntington’s.
“We are grateful to CIRM for validating our scientific approach and funding the development of this life-changing therapy for HD [Huntington’s disease] patients,” Peng Yue, PhD, co-founder and CEO of Revir, said in a company press release. “This grant will help us advance our dual HTT-PMS1 program toward clinical trials, potentially transforming the treatment landscape for Huntington’s disease patients.”
Huntington’s is caused by excessive repeats of a sequence of three DNA building blocks, CAG, in the HTT gene, leading to the production of an abnormally long version of the huntingtin (HTT) protein that is prone to form toxic clumps in nerve cells. Although the inherited number of CAG repeats is fixed at birth, some brain cells accumulate further repeat expansions over time, a process called somatic CAG repeat expansion. These somatic expansions are believed to contribute to disease onset and progression, with longer CAG repeats being associated with earlier onset and more severe disease.
Treatment aims to slow disease progression
Available treatment options can help to ease symptoms and improve quality of life, but no approved Huntington’s medication can slow or stop the disease’s progression.
Revir is developing an oral therapy that will promote the selective destruction of messenger RNA (mRNA), the molecule derived from DNA that guides protein production, of both mutant HTT and PMS1, a protein that promotes somatic CAG repeat expansion.
By lowering the production of both these proteins, the therapy is expected not only to reduce the toxic aggregates of mutant HTT that drive neurodegeneration but also to suppress further CAG repeat expansion, potentially slowing disease progression.
The CIRM grant, awarded through a competitive peer review process, will help the company advance its candidate through rigorous preclinical studies, which are necessary before testing the therapy in humans, and into clinical trials. The initial goal is to ensure that the treatment is effective in slowing disease progression and is appropriate for routine clinical use.
“By leveraging our proprietary RNA-targeting technology and our platform VoyageR, this molecule is poised to revolutionize how we treat HD — offering not just symptomatic relief but a potential disease-modifying therapy, filling an unmet medical need,” said Paul R. August, PhD, Revir’s chief scientific officer. “The ability to deliver this therapy orally would mark a significant breakthrough, enhancing HD patient compliance and quality of life.”
Revir’s VoyageR platform uses artificial intelligence to rapidly identify therapeutic targets and promising lead mRNA-targeting therapy candidates.
The company said advancement of its Huntington’s therapy candidate will pave the way for the development of similar candidates designed to treat other genetic disorders.
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