Trial of high-need gene therapy set to restart following the deaths of several participants and the lifting of a clinical hold imposed by the US FDA last year.

Audentes Therapeutics is aiming for regulatory submissions next year for its lead product, a skeletal muscle-targeted gene therapy, which continues to show promise in X-linked myotubular myopathy.

Novartis and Roche have announced additional interim clinical data as their candidate spinal muscular atrophy therapies near the marketplace.

Vanderbilt launches new biotech with Deerfield aimed at reworking the drug development paradigm, while also expanding its cancer research work with BI. Grunenthal partners with Katholieke Universiteit Leuven to investigate ion channel modulation approaches in pain.