miRecule At Precipice of Breakthrough Treatment
A St. Bonaventure University alumnus who committed his career to finding a solution for a rare condition that runs in his own family said his startup company is on the brink of a game-changing option, not just for those who live with one disease, but for others.
Anthony Saleh, Ph..D., CEO of the Brown and White Ventures-backed company miRecule, shared the latest on the Bethesda, MD pharma group on the Brown and White Ventures podcast.
"I think we’re really at the precipice of seeing what we set up," said Saleh. "Hopefully very soon we’re gonna see the drug actually start to treat patients and we’re gonna see if that is safe for patients."
miRecule is a biotechnology company focused on developing RNA therapeutics for diseases that lack existing treatments or cannot be addressed by traditional small molecule or biologic drugs.
RNA acts as an intermediary molecule that reads genetic information from DNA to create proteins. By intervening in this information flow, miRecule aims to correct mutations or repress inappropriate gene expression that causes diseases.
Saleh's company focuses on rare, severe muscle diseases, including Facioscapulohumeral muscular dystrophy (FSHD) and centronuclear myopathy (CNM).
Saleh’s interest in healthcare and science began as an undergraduate student at St. Bonaventure University (2001), where he studied biology and biochemistry. His commitment to this field became personal after learning that FSHD, one of the two diseases miRecule is working to treat, runs in his own family.
"Once we show that the drug is safe, then we can start to ask is it actually working is an FSHD patient not getting worse are they able to maintain muscle strength muscle mass and that would be in a really exciting to see that happen," said Saleh.
After miRecule meets that milepost in its journey, Saleh said the company will launch its own program for centronuclear myopathy next.
"We’ve now identified what we think is going be the compound that eventually goes into people. We made a first submission to the FDA," said Saleh, who has worked with other startups, and before that, conducted research at Johns Hopkins University and the National Institutes of Health.
miRecule's FSHD program has been licensed to pharmaceutical partner Sinofi. It is preparing for human clinical trials and preparing for a fundraising round to meet the next step in its journey.
"We’re looking to raise money right now so that we can do all the required testing for the FDA to show that we manufactured the drug properly that it can go into patients and that it’s safe enough."
Learn more about mirecule and Anthony Saleh at the Brown and White Venturess Podcast
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