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.

Over the next three months, the agency could approve a rival to a fast-selling Pfizer heart drug, a much-debated lung cancer medicine and an addition to Vertex's dominant cystic fibrosis business.

The agency will offer earlier meetings with drugmakers as well as solicit more patient feedback on the process.

Biosimilar competition to aging blockbusters will erode a large chunk of the pharma giant’s top line over the next few years.

As CVS pursues a massive cost-cutting plan — and reportedly considers a potential breakup of its businesses — the healthcare behemoth has decided to cut 1% of its workforce.

The company will permit six generic drugmakers to make and sell lenacapavir in 120 countries that have high incidence of the disease, but limited resources.

Having already been on a winding journey, Enjaymo, which Sanofi acquired through a 2018 buyout of Bioverativ, is now headed to Recordati as part of a deal announced Friday.

Medical meetings often feature important clinical trial results, making them barometers of biotech and pharma companies' research progress. Here’s a list of conferences to watch in 2025.

The official launch comes five months after a filing indicated major investors such as Atlas Venture and Bain Capital were backing the biotech, then named “Hercules CM NewCo.” ...