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 ...

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 ...

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.

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

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

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

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 ...

When gene therapy was first proposed in the 1970s, the idea seemed straightforward but revolutionary: replacing defective genes with functional ones to cure a disease. But over the decades, this ...

Please provide your email address to receive an email when new articles are posted on . An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing ...