Phase 3 findings could position the biotech’s therapy to become part of a “new standard” for SMA, its CEO said, while boosting research into whether it can preserve muscle in people with obesity.

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

Producing safe, effective, high-quality viral vector therapeutics depends on a robust and tailored analytical testing strategy.

The Cambridge, Massachusetts-based biotech is using what it calls ligand-siRNA conjugates to reach drug targets in the kidney, which historically have been difficult to reach with RNA therapies.