In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

Showcased 7 presentations at ISSCR 2025, including a featured oral presentation on first-in-human gene therapy for heart ...

RNA gene scissors (CRISPR-Cas13) are gaining significant attention as a next-generation gene therapy with fewer side effects.

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

Greater numbers of circulating stem cells increase the efficacy of in vivo gene therapy techniques, potentially reducing the ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, manage, and potentially cure a wide range of inherited, rare, and acquired ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...