A University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a ...

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 ...

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.

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

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

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 ...

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, ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

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 ...