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

When even one tiny section of a person’s genetic code becomes mutated or modified, cells can begin to act out of ...

Additionally, improving CAR-T biological efficiency could reduce dosing needs, further cutting costs and expanding access.

The FDA granted regenerative medicine advanced therapy designation to ATSN-201, a gene therapy candidate for the treatment of ...

AMT-130 is administered through magnetic resonance imaging-guided, convection-enhanced stereotactic neurosurgical delivery into the striatum.

Despite the challenges, including what CSL CEO Paul McKenzie has described as a fragmented U.S. healthcare system, the blood ...

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.

Haemophilia, a rare bleeding disorder, once drastically shortened lifespans. Prophylaxis, involving regular clotting factor ...

According to estimates by the World Federation of Hemophilia, nearly 80% of hemophilia cases in India remain undiagnosed, ...

UMass Chan Medical School has laid off or furloughed 200 employees, as the campus grapples with cuts to NIH research and ...

Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...