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6
Nov
2024
Developing a New Class of RNA Therapeutics: Leslie Williams on The Long Run
Today’s guest on The Long Run is Leslie Williams.
Leslie is the co-founder and CEO of Boston-based hC Bioscience.
Her company is working to develop a new class of RNA medicines directed at transfer RNAs, also known as tRNA. The gist of the idea is to make these tRNA therapies, delivered with a lipid nanoparticle, that can carry instructions to overwrite a premature termination codon. The premature termination codons, or PTCs, stop the cell from producing certain full-length proteins. By overwriting the PTC, the hope is to coax the body to produce full-length, functioning proteins.
You could think of it like gene therapy in its ability to tackle disease at the molecular roots, but without the risk associated with viral vectors, and with the potential to re-dose the drug if the effect wears off over time. Those who want to dig into the science can read a seminal 2019 paper in Nature Communications, from a team at the University of Iowa.
In theory, this class of medicines has broad potential. An estimated 10-15 percent of all inherited diseases are caused by premature termination codons. hC Bioscience has lead programs for hemophilia A and Duchenne Muscular Dystrophy. Clinical trials are scheduled to begin in late 2025, and the company hopes to have the first batch of results from patients in early 2026.
One of the key questions for hC Bioscience and others in the field is whether they can deliver enough of the therapy to the relevant cell types to produce enough of the desired proteins to make a difference.
This episode includes some revealing insights about who Leslie is, based on her upbringing in a small town in Iowa, and how, over time, she gained skills and experiences that got her to where she is today.
Please enjoy this episode with Leslie Williams.