Vectorology - GEG Tech top picks

A child with mucopolysaccharidosis type IIIA (MPS IIIA) has died in Lysogene’s pivotal trial for its lead gene therapy. LYS-SAF302 is designed to deliver a functional copy of the SGSH gene directly to brain cells using the adeno-associated virus carrier, AAVrh10.

As Solid’s repeated trial suspensions have illustrated, safety fears continue to plague gene therapy developers relying on high doses of AAV vectors to deliver their supposed one-time cure. More recently, Astellas subsidiary Audentes reported the third death in its trial for X-linked myotubular myopathy, highlighting trouble ahead for a booming field that’s also clouded by durability and CMC challenges.