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Cetya Therapeutics Announces STTR Grant Award

$240,000 Grant will allow evaluation of Cetya’s HDAC inhibitors for treatment of sickle cell disease

Cetya Therapeutics, Inc. announces the award of a $240,000 Phase I STTR grant over a 12-month period to support the in vitro assessment of its HDAC inhibitors for upregulation of fetal hemoglobin as a potential treatment modality for sickle cell disease. Much research over the past few decades has shown that even small increases in the level of fetal hemoglobin can mitigate the severity of symptoms associated with sickle cell disease. Drug intervention that aims to increase expression levels of the fetal-globin chain, and thus fetal hemoglobin, is one potential therapeutic approach to reduce the underlying pathology and provide clinical benefit to these patients. Sickle cell disease and related conditions, thalassemias, are among the most common inherited genetic disorders globally, affecting millions of people. 7% of the world’s population carries an abnormal hemoglobin gene, 400,000 newborns are born annually with a severe hemoglobinopathy. Sickle cell disease (SCD) affects 100,000 individuals in the US, who require $3B in annual medical care costs. These diseases result from a mutation in the beta-chain of hemoglobin A that causes a reduced or abnormal hemoglobin protein. Fetal hemoglobin, another normal type that is produced during fetal life but downregulated shortly after birth, is found in everyone, including patients with sickle cell disease and the thalassemias, and can compensate for deficient or defective forms. It is thus a natural remedy – but requires activation. HDACs (histone deacetylases) are a family of enzymes that are important in regulating expression of many genes, which includes fetal-globin. Class I HDACs are components of the repressor complexes that are responsible for the down-regulation of fetal-globin. Thought leaders believe that Class I HDAC inhibitors may have application in treating hemoglobinopathies such as sickle cell disease.

“I am excited by the potential of Cetya’s portfolio of HDAC inhibitors based on the early data that I have obtained. It will be very encouraging if those initial results translate to sickle cell patient samples to be examined under this STTR grant”, stated Dr. Susan Perrine of the Boston University School of Medicine and co-Principal Investigator for the grant along with Dr. Robert Williams of Colorado State University.

Largazole analogs represent an opportunity to develop HDAC inhibitors that may mitigate dose-limiting toxicities and serious adverse events that have plagued HDAC inhibitor drug development, with the potential to address therapeutic areas outside of oncology, such as sickle cell disease. Cetya believes its proprietary platform is positioned to overcome these limitations. Cliff Hendrick, CEO of Cetya, stated, “The STTR grant with Dr. Perrine is a tremendous opportunity for Cetya to showcase its HDAC inhibitor portfolio and gain supporting evidence that these analogs might be useful outside of the currently approved HDAC inhibitor indications within oncology.” Cetya has the exclusive worldwide rights to the histone deacetylase (HDAC) inhibitor patent estate developed in the laboratory of Dr. Williams, University Distinguished Professor of Chemistry at Colorado State University. One of the co-inventors on the IP developed by Dr. Williams is Dr. James E. Bradner, formerly of the Dana Farber Cancer Institute, and now President of Novartis Institutes of Biomedical Research. Dr. Bradner, along with another collaborator of Dr. Williams, Dr. Stuart Schreiber of the Broad Institute, are recognized leaders in understanding the structure and function of HDACs, and in the design of isoform-specific inhibitors of HDACs.



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