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

And more pharma news updates from Pharmalittle.

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

BIRAC and Miltenyi Biotec India have joined forces to elevate India's standing in cell and gene therapy (CGT). The ...

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 ...

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

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 ...