Just a few weeks after closing a $155 million series B, RNA editing biotech AIRNA is bringing an experienced gene therapy ...

The prospects for a one-time treatment that reduces the risk of heart disease for the rest of a person’s life just got ...

Getting therapeutic drugs past the blood-brain barrier has long been a major challenge in treating brain diseases. Now, researchers have explored how cholesterol-modified heteroduplex oligonucleotides ...

Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of limb-girdle muscular dystrophy (LGMD), a group of rare genetic disorders marked ...

Wall Street analysts said the results for Verve’s therapy appeared competitive on efficacy, while avoiding any major safety ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

The law firm of Kessler Topaz Meltzer & Check, LLP ( is currently investigating potential violations of the federal ...