The clinical development head of Novartis Gene Therapies talks about the progress for SMA patients more than seven years after treatment began, and hopes of boosting outcomes by treating pre-symptomatic patients.

Siegall is moving on after an unhappy exit from Seagen with a new role at another company with a novel targeted oncology platform.

ChatGPT’s ability to perform sophisticated analysis and writing tasks has taken the world by storm, and now one developer has launched its own AI aimed at revolutionizing pharma’s business intelligence functions.

New long-term data from Novartis’s Zolgensma and Roche’s Erysdi will help close the gap on Biogen’s market leader Spinraza, but the three therapies look set to develop distinct or even complementary roles as the market matures.

More than one year into the conflict, pharma employees in Ukraine have made heroic efforts to maintain existing clinical studies – but what the country’s doctors and CROs really want is for international companies to start opening new trials again.

The route to market for Sarepta’s gene therapy candidate SRP-9001 for Duchenne muscular dystrophy has just got more complex after a U-turn by the US FDA. Its accelerated approval will now be scrutinized by outside experts – but the company remains confident in its data.