In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

There is no cure for Huntington's disease, but a new gene treatment is the first to significantly slow the symptoms in human trials.

Retiree Chip Hailey, who has a severe form of hemophilia B, participated in a groundbreaking gene therapy trial. Hailey experienced a life-threatening delay in treatment after a car accident, ...

Over the last 15 years, I have witnessed how cell and gene therapy (CGT) has evolved in many ways, spanning from research and development to manufacturing. Breakthroughs such as CAR-T and gene therapy ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast ...

Four patients with infantile-onset Pompe’s disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid ...

The world's first patient to be treated with personalized gene editing therapy is finally headed home after over 300 days in the hospital. Nine-month-old KJ Muldoon recently underwent CRISPR-based ...