Phase 2/3 Trial for MPS IIIA Gene Therapy Candidate LYS-SAF302 Starts Dosing Patients
The first patient has been dosed in a Phase 2/3 clinical trial evaluating Lysogene‘s gene therapy candidate LYS-SAF302 for Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA). LYS-SAF302 is a viral vector that carries a healthy SGSH gene. This gene gives instructions to produce an…