New Jersey-based pharmaceutical company PTC Therapeutics, Inc. has received marketing authorization from the European Commission for a gene therapy treatment manufactured in partnership with MassBiologics of UMass Chan Medical School in Worcester.
The treatment, Upstaza, is the first approved gene therapy approved for treatment developed for aromatic L-amino acid decarboxylase deficiency, the school announced on Aug. 2.
AADC, according to the medical school, is a very rare genetic disorder impacting babies from the first months of life and shortens the lifespan of children who have it.
MassBiologics provided contract manufacturing, process development, and registration support for Upstaza, according to UMass Chan.
