HNF is Reshaping Drug Repurposing for 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.
HNF is advancing one of its most promising Therapeutic Research in Accelerated Discovery (TRIAD) initiatives: repurposing existing FDA-approved drugs for Charcot-Marie-Tooth disease (CMT), a hereditary peripheral nerve disorder. We are asking for your help as we move into the next stage of this work.
Where We Are Now
HNF has completed the Discovery stage of this initiative, identifying dozens of potential drug candidates across several CMT subtypes. We are now entering the Preclinical stage, during which candidate drugs will be further tested for activity in laboratory cellular models of CMT.
Our Partner: Rarebase
HNF has partnered with Rarebase, a public benefit precision medicine company. Rarebase has screened a large library of FDA-approved small molecules using its technology platform, called Function™, to identify candidates for various CMT types. Because many CMT subtypes have well-documented genetic causes and disease mechanisms, HNF and Rarebase are able to target the genetic root cause of the disease rather than just managing symptoms.
Why Drug Repurposing Matters
Developing an entirely new drug can cost tens to hundreds of millions of dollars and take an average of 10 to 17 years to reach patients. Published reports suggest that repurposed drugs may be approved in an average of 3 to 12 years, potentially reaching patients much sooner. HNF is excited about this strategy for accelerating access to new treatments.
HNF thanks all the patients who provided blood samples to help develop the cellular models needed for this phase of research.
How You Can Help
As a CMT patient or caregiver, your participation in research is essential. Join GRIN, HNF’s patient registry, to contribute to this and future studies: learn more about GRIN.