The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Intellia Therapeutics’ stock may have taken a hit in the wake of details of a grade 4 adverse event in a phase 3 trial, but ...

The FDA has slapped a clinical hold on Rocket Pharmaceuticals’ pivotal gene therapy trial in response to a death. Rocket, ...

Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of ...

Deepa Soundara Rajan, MD, and Jennifer L. Gannon, MD, discuss the current state of gene therapy for treating neurologic ...

Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment May 15, 2025 — A research team has developed and safely delivered a personalized gene ...

Find out if gene therapy has also find its way into skin diseases. With two approvals in the last 2 years, is it the start of ...

A personalized gene editing therapy was successfully used to treat an infant with CPS1 deficiency, a disease that causes ...

But, in some ways, he’s lucky. In June, the Food and Drug Administration expanded the use of Elevidys, a gene therapy, removing some of the previous restrictions to receive the treatment.