The Hereditary Neuropathy Foundation Announces New Partnership With Acetylon Pharmaceuticals To Explore Potential Therapies For Charcot-Marie-Tooth Disease

by | Apr 6, 2016 | 5 comments

The Hereditary Neuropathy Foundation (HNF) is proud to announce a new partnership with Acetylon Pharmaceuticals, Inc., a leader in the development of selective histone deacetylase (HDAC) inhibitors for enhanced therapeutic outcomes.

As a supplement to HNF’s long-standing collaboration with University of Sheffield UK, Acetylon will provide an HDAC6 inhibitor compound for testing in a preclinical model of advanced therapies for Charcot-Marie-Tooth (CMT), the most common inherited peripheral neuropathy.

To date, HNF has committed $1.5 million to CMT research as part of their Therapeutic Research In Accelerated Discovery (TRIAD) program, which fosters collaboration among academics, government, and industry to accelerate potential treatments for CMT.

In addition to the Acetylon partnership, HNF will continue to approve expenditures to expedite the search for treatments and cures. Dr. Andy Grierson, a group leader from the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield UK, has developed a zebrafish model of CMT2A, the fifth most common form of CMT. He is using this model as a platform to test therapeutic compounds that could lead to potential treatments for CMT patients.

Dr. Andy Grierson, a group leader from the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield UK, has developed a zebrafish model of CMT2A, the fifth most common form of CMT. He is using this model as a platform to test therapeutic compounds that could lead to potential treatments for CMT patients.

Sean Ekins Chief Science Officer of HNF states: “HNF recognizes the sense of urgency to get treatments to patients and families as quickly as possible. Whenever possible, we try to connect companies with researchers who are pushing the envelope and trying to find potential treatments for CMT. Whether it’s a focus on drug development, high throughput screens with FDA approved drugs, or novel compounds that could lead to new targets, we are constantly searching for ways to help accelerate this process.”

Allison Moore Founder and CEO of HNF states: “In addition, HNF continues to support the development of their patient-centered programs and support clinical trials. We are committed to expanding our programs to facilitate the long process of therapy development by identifying new endpoints for clinical trials, providing robust patient data, supporting regulatory processes, and completing human trials.”


 SITraN is a world-leading research center at the University of Sheffield, purpose-built and dedicated to research into neurodegenerative diseases. The state-of-the-art research facility was opened in 2010 by HM The Queen and uniquely allows the multidisciplinary collaboration of clinicians, scientists, and health professionals to develop new treatments for the benefit of patients.



Acetylon Pharmaceuticals, Inc., based in Boston, Massachusetts, is a leader in the development of novel small molecule drugs targeting epigenetic mechanisms for the enhancement of therapeutic outcomes in cancer and other critical human diseases.

The company’s epigenetic drug discovery platform has yielded a proprietary portfolio of optimized, orally-administered Class I and Class II histone deacetylase (HDAC) selective compounds. Alteration of HDAC regulation through selective HDAC inhibition is thought to be applicable to a broad range of diseases including cancer, sickle cell disease and beta-thalassemia, and autoimmune and neurodegenerative diseases.

Acetylon’s lead drug candidate, ricolinostat (ACY-1215), is a selective HDAC6 inhibitor currently in Phase 2 clinical development for the treatment of multiple myeloma. In 2013, the company announced a strategic collaboration agreement with Celgene Corporation, which includes an exclusive option for the future acquisition of Acetylon by Celgene.

Acetylon’s scientific founders are affiliated with Harvard University, the Dana-Farber Cancer Institute, the Massachusetts General Hospital, and Harvard Medical School.


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  1. Ronald Riskevich

    I am a CMT Patient. I’m interested in clinical trials. Keep me informed.



  2. Kieran Schaefer

    Please send me news letter

    • courtney

      Hi Kieran,

      You are subscribed to the newsletter.

  3. Ronald

    I am a CMT patient. I am interested in clinical trials.


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