Gene Therapy LYS-SAF302 Boosts or Stabilizes Development After 2 Years

Children with Sanfilippo syndrome type A, including those with severe disease, showed stable or continuously increasing cognitive, language, and motor functions two years after dosing with the investigational gene therapy LYS-SAF302, new data from the AAVance trial showed. The therapy also maintained low levels of the disease-related biomarker heparan sulfate…

Lysogene Regains Rights to LYS-SAF302 for Sanfilippo Type A

Lysogene is terminating its licensing agreement with Sarepta Therapeutics for LYS-SAF302, its investigational gene therapy for Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA). The therapy is being evaluated in children with the condition in a Phase 2/3 clinical trial called AAVance (…

Lysogene Announces Goals for Its MPSIIIA Clinical Program

After a pivotal start in 2019 to its clinical program for Sanfilippo syndrome type A, Lysogene has announced its new goals for developing a treatment for the disease, also known as mucoplysaccharydosis IIIA (MPSIIIA). During the first half of this year, Lysogene has achieved important milestones in…