Revir raises $30M to develop oral RNA-targeted therapies
Its AI platform may be used to target many proteins considered 'undruggable'
The biotechnology company Revir Therapeutics has raised $30 million in funding to advance the development of oral genetic therapies for amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders.
This Series A financing was led by the healthcare-focused venture capital firm Lapam Capital. Revir will use the proceeds to identify additional small molecules that can reduce the production of disease-causing proteins by targeting their messenger RNA, which are the intermediate molecules originating from DNA that are used as templates for protein synthesis.
By focusing on RNA, the approach may be used to target many proteins that were so far considered undruggable — either due to lack of easy-to-access crevices that standard drugs bind to, being located in regions within cells that make them difficult to access, or having similarities with other molecules that could increase the risk of off-target effects.
80% of all known disease-related proteins ‘undruggable’
It’s estimated 80% of all known disease-related proteins fall into this “undruggable” category.
“Neurological diseases affect a large number of individuals worldwide, many of which do not have disease modifying therapies available,” Peng Yue, PhD, co-founder and CEO of Revir, said in a press release. “ReviR is dedicated to developing accessible, safer genetic therapies that can be administered orally.”
In most ALS cases, abnormal proteins tend to accumulate and form clumps inside nerve cells, causing significant damage that leads to the progressive symptoms that characterize the disease.
Reducing the production of these proteins could slow or stop ALS progression, but the vast majority of disease-driving proteins cannot be targeted with common treatments.
To address that, Revir is developing small molecules that can be taken orally and reduce those proteins by targeting their messenger RNA molecules, which are needed for the cell’s protein-making machinery to synthesize a given protein.
During protein production, RNA is matured through a process known as RNA splicing, in which parts of the RNA are removed to produce the final set of instructions for the protein. Revir’s small molecules are designed to modify the splicing process, which creates mature messenger RNA molecules than cannot give rise to the disease-causing protein.
Revir’s small molecules created with AI-driven drug discovery platform
The company’s small molecules are created with its artificial intelligence (AI)-driven drug discovery platform, Voyager, which first uses AI and machine learning to design many RNA-targeting compounds that modulate splicing, and then runs computational analyses to determine which of those small molecules can potentially modulate disease progression and via which disease targets.
The platform can then be used to design an oral compound containing the identified small molecule for further studies. These less invasive genetic therapies are expected to be more patient-friendly than conventional RNA-targeted therapies.
“Small molecule targeting RNA represents a highly challenging and innovative field,” said Zhiahu Yu, founding partner of Lapam Capital. “RNA targets offer greater potential compared to protein targets, but also present higher development difficulties. We have great confidence in ReviR’s platform, which can identify potential RNA targets and efficiently develop safe and effective RNA-targeted therapies. This enables the development of orally administrable small molecule drugs for previously undruggable targets.”
In addition to Lapam, existing investors CDH Investments, 5Y Capital, and Yael Capital, and new investors, including Xtalpi and the CMT Research Foundation, also participated in the financing round.
“We value the support from new and existing investors as we progress from initial platform development to clinical trials, continually enhancing our AI platform and advancing our pipeline,” Yue said.
In addition to ALS, the Voyager platform will be used to develop RNA-targeting treatments for other neurological diseases, including Huntington’s disease and Charcot-Marie-Tooth disease.
About the Author
