New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

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

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 ...

Universal gene‑edited CAR‑T therapies turn donor T cells into off‑the‑shelf cancer treatments, cutting delays and costs while ...

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.

But cell and gene therapies (CGTs) are catching up. During Cell and Gene Therapy International Europe 2025, held in Berlin, Germany, two prominent showcases in neurobiology have shown promise toward ...

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 ...

As CRISPR moves from lab success to real-world medicine, 2026 will test whether these revolutionary gene-editing therapies can reach the patients who need them most or remain costly, complex ...

Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting friends. It was a contrast to how Cole, 18, spent part of the 2024 holiday ...

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

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