The trial provided Class IV evidence that VY-AADC01 and the associated surgical delivery procedure were well-tolerated in patients aged 40 to 70 years with moderately advanced Parkinson disease.
In addition to the uplifted phase 1 study, Novartis also initiated the phase 3 STEER study, which will evaluate OAV-101 in treatment-naive patients with SMA type 2 aged between 2 and 18 years old.
The safety profile of the SLC13A5 vector was “excellent” according to study authors, with no adverse effects on weight, general activity, or survival in knockout mice and wild type littermates.
In preclinical data, VY-HTT01 showed a robust and durable reduction of huntingtin mRNA and protein while distributed across core areas of Huntington disease pathology.