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.

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

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

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

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

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...

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

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 ...

A Johnson & Johnson gene therapy in development for an inherited vision-loss disorder has failed a Phase 3 study, the latest clinical research setback for a rare eye disease that so far has no ...