Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to a well-tolerated safety profile and what analysts believe “sets a new bar” in terms of efficacy for the condition.

Despite securing a much-needed $5 million lifeline from an equity offering this week, HCW Biologics is continuing to warn that its financial future remains in doubt.

After more than two decades as a Big Pharma cancer leader, Nicholas Botwood is swinging into the biotech world as the new chief medical officer and R&D head of Syndax Pharmaceuticals. Botwood spent the last decade at BMS, most recently serving as head of worldwide medical oncology, and, before that, he spent 11 years as an oncology development leader at AstraZeneca.

An infant named KJ has made history as the first patient to be treated with an in vivo CRISPR gene editing therapy designed specifically for him.

This week on "The Top Line," Fierce Pharma breaks down President Donald Trump’s executive order targeting drug prices and what it could mean for the industry as broader policy shifts unfold.

With two concurrent FDA approvals, Incyte’s PD-1 inhibitor Zynyz has redeemed itself in anal cancer four years after an initial snub from the agency.

The sale allows WuXi Biologics to solely focus on its Singapore capacities while marking the first step in Terumo's global expansion.