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Mount Sinai Grants Exclusive License to Plexcera Therapeutics to Develop Treatments for Farber Disease and Cystic Fibrosis
Date:9/11/2013

New York, NY (PRWEB) September 11, 2013

Mount Sinai Innovation Partners (MSIP), part of the Icahn School of Medicine at Mount Sinai, has granted an exclusive license to Plexcera Therapeutics, LLC to commercially develop recombinant human acid ceramidase (rhAC) to treat diseases caused by genetic or disease-induced deficiencies in the enzyme rhAC.

Mount Sinai is promoting the scientific discoveries of its faculty by facilitating the establishment and supporting incubator companies to accelerate the discovery of treatments for devastating diseases, including those that often affect relatively small populations.

Plexcera was founded by Edward H. Schuchman, PhD, Genetic Disease Foundation, Francis Crick Professor of Genetics and Genomic Sciences at the Icahn School of Medicine, and Ivan Galanin, a pharma industry veteran and an advisor to MSIP, in collaboration with QOL Medical LLC, a specialty pharma company focused on rare pediatric diseases.

Two devastating childhood diseases are caused by recessive inherited mutations in the gene encoding rhAC: Farber disease, characterized by severe joint pain, inflammation and arthritis, and, a form of spinal muscular atrophy with epilepsy (SMA-PME), found in adolescents, and characterized by progressive muscle weakness. There is no therapy for either condition. In addition, in cystic fibrosis, excess ceramide accumulates in the lungs. Treatment with inhaled rhAC may address lung cell death, inflammation and susceptibility to infection seen in these patients.

“The name ‘Plexcera’ comes from the concept that rhAC is an enzyme with multiple uses. Farber disease is our first target,” said Dr. Schuchman. “We hope to launch a clinical trial of rhAC within the next 18 months. "

The licensed technology is based on more than 20 years of research conducted by Dr. Schuchman, who will serve as Plexcera’s Chief Scientific Officer, and Erich Gulbins, Ph
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