1st European Patient Treated in Phase 2/3 Trial of Sanfilippo Type A Gene Therapy LYS-SAF302

A Phase 2/3 trial of the Sanfilippo syndrome type A gene therapy candidate LYS-SAF302 has dosed its first patient in Europe. Called AAVance, the open-label trial (NCT03612869) is assessing the safety and efficacy of a one-time delivery of LYS-SAF302. The team intends to recruit a total of 20…

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…

Sarepta and Lysogene Sign Licensing Deal for LYS-SAF302, Potential Gene Therapy for Sanfilippo Type A

Sarepta Therapeutics has signed a licensing agreement with Lysogene for the development of investigational gene therapy LYS-SAF302 to treat mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, the companies recently announced. Under the terms of the deal, Sarepta gains exclusive commercial rights to…

Lysogene Plans Phase 2/3 Study of LYS-SAF302 Later This Year

Lysogene’s Phase 2/3 trial on LYS-SAF302 gene therapy, a clinical candidate for Sanfilippo syndrome type A, will begin in the second half of 2018 due to manufacturing delays. LYS-SAF302 is a viral vector that carries a healthy SGSH gene. This gene gives instructions to produce an enzyme called sulfamidase, which…