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 ...

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

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

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.

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other ...

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

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 ...

As CRISPR moves from lab success to real-world medicine, 2026 will test whether these revolutionary gene-editing therapies ...

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 ...

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 ...

The use of base editing to generate universal off-the-shelf CAR T cells induced durable remissions -- up to 36 months in one ...