VERO BEACH, Fla.--(BUSINESS WIRE)--

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, MPh, 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 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 Plexceras Chief Scientific Officer, and Erich Gulbins, PhD, from the Center for Medical Biotech at the University of Duisburg-Essen, Germany, who will serve on Plexceras Scientific Advisory Board. Dr. Schuchman has extensive research and development experience with these disorders and enzyme replacement therapy specifically. Dr. Gulbins has identified a central role for excess ceramide accumulation in cystic fibrosis, as well as other pulmonary diseases.

Mount Sinai has a strong track record of developing breakthrough products for rare diseases. Dr. Schuchman has worked with key scientific, clinical, and industry thought leaders for many years and can call on their expertise and commitment, said Mr. Galanin, CEO of Plexcera. The collaboration with QOL Medical gives us access to key infrastructure components such as clinical, regulatory, and manufacturing expertise.

As part of the license, Mount Sinai received equity in the new company, as well as royalties. This is the second major license agreement negotiated this year by MSIP in the field of orphan diseases, both originating from research conducted by the Department of Genetic and Genomic Sciences.

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Mount Sinai Grants Exclusive License to Plexcera Therapeutics to Develop Treatments for Farber Disease and Cystic ...

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