Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of ...

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. | A second patient has ...

May 1, 2025 — To help achieve more precise control of gene therapy, engineers have designed a new control circuit that can keep gene expression levels within a target range. The method could be ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

BIRAC and Miltenyi Biotec India have joined forces to elevate India's standing in cell and gene therapy (CGT). The ...

By Christy Santhosh and Bhanvi Satija (Reuters) -Shares of Sarepta Therapeutics slumped 36% in premarket trading on Monday after the company reported the death of a second patient in three months who ...

For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body -- a job commonly given to adeno-associated viruses (AAV).

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...