Mount Sinai Grants Exclusive License to Plexcera Therapeutics to Develop Treatments for Farber Disease and Cystic Fibrosis
Plexcera was founded by Edward H. Schuchman, MPh, PhD, Professor Professor of Genetics and Genomic Sciences at the Icahn School of Medicine.
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 of incubator companies and investing in the companies--by granting a license, taking equity, and encouraging faculty and staff to assume leadership positions--to develop solutions to devastating diseases, including diseases that often affect relatively small populations.
Plexcera was founded by Edward H. Schuchman, MPh, PhD, Genetic Disease Foundation - Francis Crick Professor 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 Plexcera's Chief Scientific Officer, and Erich Gulbins, PhD, from the Center for Medical Biotech at the University of Duisburg-Essen, Germany, who will serve on Plexcera's 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.
About Mount Sinai Innovation Partners
Mount Sinai Innovation Partners (MSIP), part of the Icahn School of Medicine at Mount Sinai, facilitates the transfer of discovery from the laboratory to the marketplace, acting as the interface with commercial entities.
MSIP is responsible for the full spectrum of commercialization activities required to bring the Icahn School of Medicine's inventions to life. These activities include evaluating, patenting, marketing, and licensing new technologies, while also negotiating agreements for sponsored research, material transfer, and confidentiality. For more information on MSIP, visit: http://www.mountsinai.org/innovation
About Plexcera Therapeutics, LLC
Plexcera Therapeutics was founded with the goal of bringing life-saving therapies to patients who have either a genetic deficiency in the acid ceramidase enzyme or a disease-driven imbalance between the enzyme and its substrate. The company is headquartered in Vero Beach, FL, with facilities in New York City. For more information on Plexcera Therapeutics, please visit: http://www.plexcera.com.
About QOL Medical LLC
QOL Medical LLC, based in Vero Beach, is a specialty biopharmaceutical company focused on pediatric products for genetic and rare orphan diseases. QOL invests in clinical stage and marketed products and currently markets 2 approved products: Sucraid (sacrosidase) Oral Solution for Congenital Sucrase-Isomaltase Deficiency and Ethamolin (Ethanolamine Oleate) Injection 5% for bleeding esophageal varices.
About The Mount Sinai Medical Center
The Mount Sinai Medical Center encompasses both The Mount Sinai Hospital and Icahn School of Medicine at Mount Sinai. Established in 1968, the Icahn School of Medicine is one of the leading medical schools in the United States, with more than 3,400 faculty in 32 departments and 14 research institutes. It ranks among the top 20 medical schools both in National Institutes of Health (NIH) funding and by U.S. News & World Report. The Mount Sinai Hospital, founded in 1852, is a 1,171-bed tertiary- and quaternary-care teaching facility and one of the nation's oldest, largest and most-respected voluntary hospitals. The Mount Sinai Hospital is nationally ranked by U.S. News & World Report as one of the top 25 hospitals in 7 specialties based on reputation, safety, and other patient-care factors.
For more information, visit Mount Sinai on the web, Facebook, Twitter or YouTube.
About the Mount Sinai Health System
Mount Sinai Health System is one of the largest academic medical systems in the New York metro area, with 48,000 employees working across seven hospitals, more than 400 outpatient practices, more than 600 research and clinical labs, a school of nursing, and a leading school of medicine and graduate education. Mount Sinai advances health for all people, everywhere, by taking on the most complex health care challenges of our time—discovering and applying new scientific learning and knowledge; developing safer, more effective treatments; educating the next generation of medical leaders and innovators; and supporting local communities by delivering high-quality care to all who need it.
Through the integration of its hospitals, labs, and schools, Mount Sinai offers comprehensive health care solutions from birth through geriatrics, leveraging innovative approaches such as artificial intelligence and informatics while keeping patients’ medical and emotional needs at the center of all treatment. The Health System includes approximately 9,000 primary and specialty care physicians and 10 free-standing joint-venture centers throughout the five boroughs of New York City, Westchester, Long Island, and Florida. Hospitals within the System are consistently ranked by Newsweek’s® “The World’s Best Smart Hospitals, Best in State Hospitals, World Best Hospitals and Best Specialty Hospitals” and by U.S. News & World Report's® “Best Hospitals” and “Best Children’s Hospitals.” The Mount Sinai Hospital is on the U.S. News & World Report® “Best Hospitals” Honor Roll for 2025-2026.
For more information, visit https://www.mountsinai.org or find Mount Sinai on Facebook, Instagram, LinkedIn, X, and YouTube.
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