HNF is leading one of the first research initiatives to tackle multiple types of CMT in a single project. To advance therapy development for most types of CMT, HNF launched a drug repurposing program in 2022 to bring treatments to patients faster.
Rarebase is a public benefit biotech company focused on accelerating therapy development for rare diseases through its tech-enabled drug discovery platform, Function. Using repurposed FDA-approved drugs, Rarebase works to deliver accelerated, off-label treatments for various types of CMT. It has completed the discovery phase and is entering preclinical validation of the drug candidates identified for several CMT subtypes.
Rarebase’s Function platform has enabled us to test approximately 4,000 small molecules to measure changes in gene expression for CMT. These genetic screens produced drug candidates for 10 subtypes of CMT. HNF and Rarebase have prioritized testing the most promising candidates for these subtypes (select one to learn more):
- PMP22 duplication (CMT1A)
- PMP22 deletion (HNPP)
- MFN2 (CMT2A)
- C12orf65 / MTRFR (CMT6)
- GDAP1 (CMT4A)
- CNTNAP1
Milestones
- Phase I (2022), Discovery: drug-candidate hits identified. Completed.
- Phase II (2022 to 2023), Develop CRISPR assays for CMT subtypes to test the top candidates. In progress.
- Phase III (2023 to 2024), Conduct clinical trials on the optimal candidates.
Have your voice heard by CMT researchers. Joining GRIN, HNF’s patient registry, adds the real-world data that helps prioritize and design studies like this one.