A new portable gene therapy device could allow hospital pharmacies to create personalised medicines on demand, a new study says. Rare diseases affect more than 300 million people worldwide - 36 ...

Bootstrap Bio has hired a chief science officer, opened a lab and sought seed funding to edit human cells that can be passed to future generations ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, the agency is investigating two Duchenne muscular dystrophy patient ...

Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with genetic diseases?

Google's DeepMind launches AlphaGenome, an artificial intelligence model that predicts gene expression from DNA sequence.

In this study of an AAV9-based gene therapy in four patients with infantile-onset Pompe’s disease, one patient died and one had pneumonia that led to the temporary administration of oxygen ...

Now Google’s DeepMind division says it’s made a leap in trying to understand the code with AlphaGenome, an AI model that predicts what effects small changes in DNA will have on an array of ...

Many DNA sequences, for example, influence gene activity by altering a chromosome’s 3D shape, either restricting or easing access for the machinery that does the transcription.

Scientists Advance Prospects for Permanently Putting AIDS Virus into Dormant State Using Gene Therapy 06/25/2025 ...

FDA evaluating further regulatory actions after deaths Elevidys is the only gene therapy for Duchenne muscular dystrophy Sarepta suggested updating Elevidys' label June 24 (Reuters) - The U.S ...

Sangamo's investigational gene therapy has been tied to an improvement in kidney health deterioration at 52 weeks for patients with Fabry disease.