Robert J. Desnick , MD, E. Premkumar Reddy, PhD, and Edward Schuchman, PhD, Elected to the National Academy of InventorsJanuary 26, 2021
Mount Sinai researchers Robert J. Desnick, MD, PhD, E. Premkumar (Prem) Reddy, PhD, and Edward Schuchman, PhD, became Fellows of the National Academy of Inventors (NAI).
The National Academy of Inventors (NAI) is a prestigious organization, comprised of international universities, and governmental and non-profit research institutes, that aims to recognize and encourage inventors while enhancing the visibility of academic technology and innovation. The NAI Fellows Program recognizes pioneers in a specific field. The organization also offers a Senior Members Program for researchers whose work is making a societal impact.
Robert J. Desnick, MD, PhD, Dean for Genetics and Genomic Medicine Emeritus, Icahn School of Medicine at Mount Sinai
Dr. Desnick’s basic, translational and clinical research led to advances in the delineation, diagnosis and treatment of lysosomal storage diseases and porphyrias (inherited disorders of heme biosynthesis, or enzyme deficiency). His research led the development of Fabrazyme, a recombinant enzyme replacement therapy for Fabry disease with Sanofi Genzyme, and Givlarri, an RNA interference therapy for acute hepatic porphyrias, with Alnylam Pharmaceuticals. He was a co-founder of Amicus Therapeutics, a biotechnology company that developed Galafold, an approved pharmacologic chaperone therapy for amenable mutations causing Fabry disease. Based on his clinical experience, he co-designed clinical trials for these, and related diseases, that lead to U.S. FDA and EMA approvals.
E. Premkumar (Prem) Reddy, PhD, Professor of Oncological Sciences, Icahn School of Medicine at Mount Sinai
Dr. Reddy has studied the molecular basis of cancer for over 40 years. He has cloned and sequenced a number of viral oncogenes and their cellular homologues, pinpointing the precise changes that cellular proto-oncogenes undergo to produce cancer-causing viral oncogenes. He was responsible for the discovery of point mutations in RAS genes, resulting in oncogenic activation. Dr. Reddy has pioneered the development of small molecule inhibitors targeted against oncogenes and cell cycle regulators for cancer therapy, several of which are in clinical trials. He is the founder of Onconova Therapeutics Inc. and the international cancer journal Oncogene.
Edward Schuchman, PhD, Genetic Disease Foundation – Francis Crick Professor and Vice Chair for Research for the Department of Genetics & Genomic Sciences, Icahn School of Medicine at Mount Sinai
Dr. Schuchman has discovered several drugs for the treatment of lysosomal storage diseases. He co-discovered the drug Olipudase alfa for the treatment of Type B Niemann-Pick disease, which is exclusively licensed to Sanofi Genzyme and recently successfully completed phase 3 clinical trials. He also discovered the use of recombinant acid ceramidase for the treatment of Farber lipogranulomatosis, and repurposed this same drug for cystic fibrosis. Both uses were licensed to Plexcera Therapeutics LLC that went on to sell this drug to a NYC-based biotechnology company Enzyvant for further development. Dr. Schuchman also co-discovered the repurposing of the drug pentosan polysulfate (PPS) for the treatment of mucopolysaccharidosis (MPS), a lysosomal storage disease that affects the skeletal system, skin, heart, brain, and other organ systems. The innovation led to exclusive licenses with Paradigm Biopharmaceuticals, Ltd. and ReqMed Company, Ltd. for further development.
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