Plexcera Therapeutics has received approval from Mount Sinai Innovation Partners (MSIP), part of the Icahn School of Medicine, to develop recombinant human acid ceramidase (rhAC) for treatment of diseases caused by genetic or disease-induced deficiencies in the enzyme rhAC.
Under the terms of the licensing agreement, Mount Sinai received equity in the new company, as well as royalties.
Currently, Mount Sinai is promoting the scientific discoveries of its faculty by setting up and supporting incubator firms to speed up the discovery of treatments for devastating diseases, including those which often affect relatively small populations.
Plexcera was established by Edward Schuchman of Genetic Disease Foundation, professor of Genetics and Genomic Sciences at the Icahn School of Medicine Francis Crick and MSIP advisor Ivan Galanin in partnership with QOL Medical, a pharma firm focused on rare pediatric diseases.
Recessive inherited mutations in the gene encoding rhAC causes two devastating childhood diseases, including Farber disease that is 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.
According to the company, there is no therapy available for either of the condition.
The company expects to launch the clinical trial of rhAC within the next 18 months.
The licensing agreement with Plexcera is the second deal signed this year by MSIP in the field of orphan diseases.
