Mount Sinai Innovation Partners (MSIP) 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. As part of the license, Mount Sinai received equity in the new company, as well as royalties.
Two 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), characterized by progressive muscle weakness. Currently there is no therapy for either condition.
The licensed technology is based on more than 20 years of research conducted by Edward H. Schuchman of the Genetic Disease Foundation, who will serve as Plexcera’s chief scientific officer, and Erich Gulbins, Ph.D., from the Center for Medical Biotech at the University of Duisburg-Essen, Germany, who will serve on the Scientific Advisory Board.
“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, chief executive officer of Plexcera. “The collaboration with QOL Medical gives us access to key infrastructure components such as clinical, regulatory, and manufacturing expertise.”