Explore the mRNA universe with Cytiva. In this series, our scientists share their journey and insights on mRNA synthesis for research purposes. And they share their advice on how to scale. Through peer-to-peer interactions, we continue to encourage the spirit of collaboration developed at a time when new modalities are being used as therapies for the first time. “When it comes to mRNA, many manufacturing investments are being made while still early in the process development workflow. So essentially, we…
Friday, June 23, 2023 Daily Archives
Sarepta wins FDA approval for DMD gene therapy
The US FDA has approved Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics’s adeno-associated virus-based gene therapy for the treatment of Duchenne muscular dystrophy (DMD). Having delayed the action date by almost a month, the US Food and Drug Administration (FDA) gave the thumbs up to Elevidys (previously known as SRP-9001) yesterday, a single-dose gene transfer therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD. Sarepta’s CEO Doug Ingram described the approval as “a watershed moment for…