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Pioneering HDAC6 therapy for CMT2A

HDAC6 inhibition is one of the most promising routes to a treatment for CMT2A. HNF was the first advocacy organization to fund this work, and has helped move it from a scientific idea toward the clinic. Here is how that story unfolded.

1. 2011 The science

   HDAC6 inhibition emerges as a CMT strategy

   A landmark study in Nature Medicine (Kozikowski, Van Den Bosch, and colleagues) shows that selective HDAC6 inhibitors can restore axonal transport in models of CMT. The scientific rationale for treating CMT2A this way is established.

2. 2016 HNF steps in

   First advocacy group to fund HDAC6 work for CMT2A

   HNF partners with Acetylon Pharmaceuticals, alongside its long-standing collaboration with the University of Sheffield, to test a selective HDAC6-inhibitor compound in preclinical CMT2A models. By this point HNF has committed $1.5M to CMT research through its TRIAD program.

3. 2018 A dedicated alliance

   Strategic alliance with StarWise Therapeutics

   HNF forms a strategic alliance with StarWise Therapeutics (founded by Prof. Alan Kozikowski) to advance a next-generation HDAC6 therapy for CMT2A (MFN2), with lead research by Prof. Brett Langley.

4. Ongoing Building the package

   From zebrafish to rat

   HNF-seeded work advances through preclinical models, from zebrafish to mouse to rat, assembling the evidence a first-in-human trial requires.

5. Now Toward the clinic

   Advancing toward a trial

   The program continues toward a first-in-human study for CMT2A.

   To confirmCurrent figures pending from HNF: total funding committed to the HDAC6 / Acetylon work and key dates, program stage, and target IND / first-in-human timing.

HNF advances therapies and the infrastructure of a cure. Whether an HDAC6 therapy can halt or partially reverse CMT2A depends on the subtype and how early it is caught; this reflects the current science honestly rather than promising a single outcome.