New Developments in Farber Disease Research

Enzyvant Sciences is a new pharmaceutical company that is the product of two companies: Roivant Sciences and Plexcera Therapeutics, LLC. They have formed this joint venture to focus on the treatment of Farber disease, one of the lysosomal diseases.

At Enzyvant Sciences, it is thought that the recombinant human acid ceramidase (rhAC) program will benefit from the continued involvement of the experienced medical and scientific team from Plexcera, while gaining the pharmaceutical development expertise and financial support of Roivant. Developed in the lab of Professor Edward Schuchman, MPh, PhD, the enzyme replacement therapy rhAC showed promising results in a mouse model of Farber disease. It was initially licensed by Mount Sinai Innovation Partners, part of the Icahn School of Medicine at Mount Sinai, to Plexcera Therapeutics, LLC, prior to the formation of Enzyvant.

rhAC is based on more than 20 years of research conducted by Dr. Schuchman and Erich Gulbins, PhD, from the Center for Medical Biotech at the University of Duisburg-Essen, Germany. Dr. Gulbins identified a central role for excess ceramide accumulation in cystic fibrosis and some other pulmonary diseases. rhAC may eventually be put to other therpautic uses in addition to Farber disease. Dr. Schuchman’s laboratory has been studying the biology of lysosomal enzymes, genes and diseases for more than 25 years, achieving a number of breakthroughs towards the development of novel therapies for lysosomal disorders. For example, Dr. Schuchman’s team was the first to isolate genes encoding lysosomal enzymes linked to Niemann-Pick disease types A and B, mucopolysaccharidosis type VI (MPS VI), and Farber disease.

Dr. Edward Schuchman said, “I am very excited about the future of rhAC. Similar to other enzyme replacement therapies, rhAC has the potential to be a transformative therapy for patients afflicted with Farber disease, a population with clear unmet medical needs.” Dr. Schuchman is Francis Crick Professor of Human Genetics and Vice Chairman of Research, Department Of Genetics and Genomic Sciences; and Professor, Department of Gene and Cell Medicine, at the Icahn School of Medicine at Mount Sinai in New York City. Dr. Schuchman will continue to play an integral role in the development of rhAC. Dr. Schuchman is on Plexcera’s Board of Directors and owns an equity interest therein.

Background information: Enzyvant Press Releasebackground informationearlier background information.