Plexcera was founded by
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
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