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 ...

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

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

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 ...

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 ...

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

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

Two variants in a single gene are implicated in almost all Alzheimer’s and nearly half of all dementia cases, according to ...

A large population-level analysis led by researchers at University College London has suggested that common variants of the ...

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 ...