Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their ...

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...

An Italian foundation that helped a young boy overcome Wiskott-Aldrich syndrome becomes the first non-profit in the world to ...

A 31-year-old Sligo man who was functionally blind has got his sight back, after being treated with a new gene therapy at the ...

On this episode, an infant with a harrowing diagnosis defies all odds after receiving a $2 million genetic treatment. Then, a ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

By Dr. Liji Thomas, MD Researchers successfully use an adeno-associated viral vector to restore brain function in a mouse ...

The AAV Innovation Summit 2025, hosted by Form Bio, will bring together top experts in gene therapy to discuss the latest ...

Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

Company shares preliminary results from Phase 1/2 clinical trial for patients with advanced Retinitis Pigmentosa (RP) ...