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.

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 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.

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