HNF is Reshaping Drug Repurposing for CMT

by | Mar 6, 2023 | 0 comments

In this first 2023 issue, we ask that you help us prepare to advance one of our important Therapeutic Research in Accelerated Discovery (TRIAD) research initiatives, “Repurposed Drugs for CMT.”

HNF has completed the Discovery stage with the identification of dozens of potential drug candidates for several types of CMT. Now we are initiating the Preclinical stage of the collaboration, during which the candidate drugs will be further tested for in vitro activity in cellular models of CMT.

HNF has partnered with Rarebase, a public benefit precision medicine company that has screened a large library of FDA approved small molecules to identify candidates for various types of CMT. Their tech-enabled drug discovery platform is called Function™. There are many published discoveries on the genetic cause of many types of CMT, including an understanding of the basic mechanism of disease and potential targets for FDA-approved drug repurposing. It is this understanding that allows HNF and Rarebase to target the genetic root cause of CMT.

Unlike developing a brand-new drug, which costs tens to hundreds of millions of dollars and on average 10-17 years to bring a new drug to market, reports indicate that repurposed drugs may be approved within 3-12 years, on average. The HNF team is excited about this promising strategy of accelerating potential treatments much faster.

HNF thanks all those patients that provided blood samples to develop the cellular models that are needed for this next phase.

As a CMT patient and/or caregiver of a loved one with CMT, we need you to participate in research by joining GRIN.

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