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 ...

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 ...

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 FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and older with Wiskott-Aldrich syndrome, a rare primary immunodeficiency. The ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of addiction from narcotics treatments, a breakthrough which could provide hope for ...

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 ...

But cell and gene therapies (CGTs) are catching up. During Cell and Gene Therapy International Europe 2025, held in Berlin, ...

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

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, ...

Impella, which J&J touts as the world’s smallest heart pump, has a range of devices used for various heart conditions. Lexeo ...

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 ...

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