Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Alzheimer’s may be driven far more by genetics than previously thought, with one gene playing an outsized role. Researchers ...

GEN’s first virtual event of 2026—we present a group of outstanding researchers and thought-leaders to discuss the latest advances and challenges in delivering genetic therapies.

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

One patient still in remission 3 years after single infusion of base-edited CAR7 T cells ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Medicaid is doing a novel payment system for the new, promising and expensive sickle cell treatment. It may become a model ...

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other parts of the brain. A new preclinical study has identified a gene therapy ap ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

Forbes contributors publish independent expert analyses and insights. Bruce Japsen writes about healthcare business and policy. More than 70% of employers and health plans expect affordability of gene ...