Gene Therapy for Dravet Syndrome Trialed in Mice; Sarepta's Reversal, and AstraZeneca's Updates

In the realm of gene therapy, there has been a flurry of activity and progress in recent weeks.

Firstly, scientists have made significant strides in the development of a potential gene replacement therapy for Dravet Syndrome. The promising results of these tests were detailed in an article published by GEN on March 19, 2025. This therapy was further discussed in this week's episode of Touching Base.

Meanwhile, Sarepta Therapeutics, a leading name in gene therapy, has been at the centre of some controversy. A patient died after treatment with Sarepta's gene therapy for Duchenne muscular dystrophy. An article about this unfortunate incident was published by GEN Edge on March 18, 2025.

In a positive turn of events, the PiggyBac Transposon System has been successfully used to create transgenic Cynomolgus monkeys. This groundbreaking achievement was reported by Julianna LeMieux, PhD, in an article published by GEN on March 26, 2025.

AstraZeneca, a pharmaceutical giant, has also been making headlines. The company has committed up to $11B+ to Chinese collaborations and a Beijing R&D hub, as reported by Alex Philippidis, PhD, in GEN Edge on March 21, 2025. This move is part of AstraZeneca's broader strategy to increase its R&D in China, with new drug, vaccine, and healthcare partnerships in the billions of dollars.

Interestingly, two gene editing companies have recently pivoted from tools to development, as reported by Alex Philippidis, PhD, in GEN Edge on March 20, 2025. The specific companies involved in this shift have not been publicly disclosed.

Moreover, new data has been revealed about a nonviral method for introducing transgenes into non-human primates. However, the company responsible for this breakthrough remains undisclosed between March 18 and March 26, 2025.

In the world of gene therapy, it seems that progress is being made, challenges are being faced, and collaborations are being forged. As these advancements continue, we can look forward to a future where gene therapy may offer hope and healing for a wide range of diseases.

Gene Therapy for Dravet Syndrome Trialed in Mice; Sarepta's Reversal, and AstraZeneca's Updates