Foundation research

ORYZON collaborates with the CMT Research Foundation in the

  • Explore Oryzon’s HDAC6 Inhibitors on Charcot-Marie-Tooth disease
  • CMTRF to finance live efficacy testing in the CMT preclinical model

MADRID and BOSTON, July 26. 12, 2022 (GLOBE NEWSWIRE) — Oryzon Genomics, SA (ISIN: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with high unmet medical need, announced today today launched a preclinical collaboration with the CMT Research Foundation (CMTRF), an American patient-led non-profit organization focused on providing treatments and cures for Charcot-Marie-Tooth disease (CMT ), to explore Oryzon’s histone deacetylase 6 (HDAC6) inhibitors.

CMT is a progressive degenerative disease involving the peripheral nerves. It affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and more than 3 million people worldwide. It is one of the most common conditions among rare diseases and currently lacks effective treatments or cures. CMT is caused by a variety of genetic mutations. CMT1A is the most common of all, accounting for about half of all people with CMT.

HDAC6 inhibitors have previously been described as potentially effective treatments for CMT. Oryzon recently completed an HDAC6 discovery program leading to the selection of two potential preclinical candidates with promising efficacy, selectivity and safety. As part of this collaboration, the CMTRF will fund a series of live tests with Oryzon’s HDAC6 candidates in a mouse model of CMT1A, which reliably recapitulates many symptoms of this disease in humans.

Dr. Jordi Xaus, CSO of Oryzon, said: “CMT is the most promising indication for our HDAC6 precandidates. CMTRF is the perfect ally to explore our compounds in this indication, as it has a close connection to the CMT patient community, a highly experienced scientific advisory body and a strong commitment to finding treatments for CMT. We are delighted to start this collaboration. If the results are positive, our HDAC6 program would be closer to clinical development and offer hope for patients with CMT. This program would become our second program in epigenetics in nervous diseases.

“We are delighted to collaborate with Oryzon on this important project,” said Cleary Simpson, CEO of the CMT Research Foundation. “HDAC6 inhibitors show promise as potential therapies for several forms of CMT, including the most common form, CMT1A. With funding from the CMT Research Foundation, Oryzon will be able to rapidly test its newly discovered HDAC6 inhibitors in CMT1A model mice, paving the way. People with CMT currently have no effective treatment or cure, and we believe this project has the potential to change that for millions of families.

The CMT Research Foundation (CMTRF) is solely focused on providing treatments and cures for CMT. Founded by two patients committed to accelerating drug delivery to people with CMT around the world, the organization funds research for drug development. The federal 501(c)(3) tax-exempt organization is supported by personal and corporate financial donations.

About Oryzon
Founded in 2000 in Barcelona, ​​Spain, Oryzon (ISIN Code: ES0167733015) is a clinical-stage biopharmaceutical company considered the European leader in epigenetics. Oryzon has one of the strongest pipelines in the field, with two LSD1 inhibitors, iadademstat and vafidemstat, in phase II clinical trials, and other assets in the pipeline directed against other epigenetic targets. Additionally, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignancies and neurological diseases. For more information visit

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