Beam Therapeutics shows promising BEACON trial results in SCD, backed by strong cash reserves and pipeline momentum. See why ...

Justin Taylor, M.D., a physician-scientist at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller ...

Eli Lilly will buy Boston-based Verve Therapeutics, Inc. in a billion dollar deal with plans to advance Verve's new line of ...

Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of ...

A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...

A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. | A second patient has ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...

Despite setbacks, Sarepta’s gene therapy pipeline and market leadership in DMD present a high-risk, high-reward setup. Read ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...

You can reach Jason on Signal at JasonMast.05. What will the Trump administration mean for gene therapy? Researchers, advocates, and executives got a glimpse of the answer Thursday, as the Food ...

For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body -- a job commonly given to adeno-associated viruses (AAV).

A new gene therapy targeting the heart protein cBIN1 reversed heart failure and restored heart function in pigs, showing unprecedented recovery and a 30% improvement in heart performance. If ...