In a phase 2 study, SAR402663 gene therapy for wet age-related macular degeneration showed positive efficacy and safety ...

The plan builds on recent FDA moves to speed "personalized" treatments by offering an approval framework for therapies ...

Cardiologists who study gene therapy explain its promise, risks and the safeguards needed for responsible use.

Sujith Ramachandran, PhD, associate director of Trinity Life Sciences, grouped access barriers into 3 buckets: upfront ...

Cell and Gene Therapies (CGTs) present unique development challenges, from novel manufacturing processes and delivery methods ...

Therapy pioneered by biotechnology Chinese firm HuidaGene showed promise in animals but caused a fatal reaction in a child.

Gene therapy cut severe infections 93% in Wiskott-Aldrich syndrome, using patients’ own modified stem cells to treat a rare inherited immune disorder.

In recent months, news came out about several distressing cases that have underscored the risks of gene therapy, which ...

“It’s something that we would have never really expected to happen in our career, for kids to be able to hear without the need of devices,” otolaryngologist Dr. John Greinwald Jr. explained ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

After decades developing gene therapies for ultra-rare conditions, Alessandra Biffi is researching how altering a patient’s ...

New data showing rapid and durable full-length MECP2 expression in vivo with single-stranded AAV support improvements ...