KJ Muldoon, an infant with a life-threatening metabolic disorder who received a pioneering gene-editing therapy at Children’s Hospital of Philadelphia, went home Tuesday after 307 days at the hospital ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

After 307 days at CHOP, KJ was dressed in what early studies suggest are the world’s smallest and cutest graduation ...

After 307 days at the Children's Hospital of Philadelphia, a local baby who was born with a rare and usually fatal disorder ...

CRSP aims to extend its gene-editing lead with in vivo candidates after Casgevy's ex vivo breakthrough success.

Baby KJ was successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of ...

Global Akeega sales will reach $676 million by 2030, while Talzenna and saruparib reach $618 million and $628 million ...

Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by synaptic dysfunction, neuronal loss and the accumulation of amyloid plaques and neurofibrillary tangles, ...

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...

Discover the major highlights from ASCO 2025, according to researchers and drug developers who attended the convention.