ARTICLE | Emerging Company Profile
Breaking down ceramide
Plexcera developing ceramidase for Farber disease, spinal muscular atrophy
November 24, 2014 8:00 AM UTC
Plexcera Therapeutics LLC is developing a recombinant human acid ceramidase as a disease-modifying therapy to treat Farber disease and a subset of spinal muscular atrophy, two ultra-rare diseases for which there are no approved drugs.
Ceramidase breaks down ceramide, which is an essential lipid that modulates signaling pathways involved in cell growth and inflammation. When ceramidase is deficient, or when a disease process results in excess ceramide production, the lipid accumulates in tissues and joints and causes apoptosis and accelerates inflammation...