Pfizer announced in February 2025 that it was taking Beqvez (fidanacogene elaparvovec), its gene therapy for hemophilia B, ...

Pfizer and BioMarin have pulled their hemophilia gene therapies, citing low demand. To prepare for his presentation at the ...

Cell and gene therapies are moving towards correcting root causes of diseases. Let's take a look at future cell and gene therapy trends.

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...

CANADA - 2025/09/24: In this photo illustration, the uniQure logo is seen displayed on a smartphone screen. (Photo Illustration by Thomas Fuller/SOPA Images/LightRocket via Getty Images) Senior ...

Enein, MD, PhD, has found himself in a win-win situation: winning not one but two awards from the American Society of Gene + Cell Therapy (ASGCT), the leading professional organization in the field.

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...