Luxturna was the first FDA-approved gene therapy for a disease caused by mutations in a specific gene. It helped establish ...

Dr. Swee Lay Thein and Dr. Stuart Orkin won the $3 million Breakthrough Prize in Life Sciences for their work toward a ...

The Breakthrough Prize Foundation awarded researchers involved in developing gene therapies for blindness and sickle cell ...

For the roughly 80 million people worldwide living with glaucoma, daily life often means watching the world slowly close in.

J&J will hand over the rights to bota-vec for $25 million upfront, clearing MeiraGTx to seek regulatory approvals in the U.S.

Detroit patients getting HEMGENIX report life‑changing results; trial data show 94% off prophylaxis at five years.

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

After its bet on a rare eye disease gene therapy ended in a phase 3 fail last year, Johnson & Johnson is now returning 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 gene therapy for deafness is helping patients hear within weeks, offering hope for children and adults with genetic ...

Roche is launching a new global phase 3 trial for the controversial Duchenne muscular dystrophy gene therapy Elevidys in ...

Scientists discover salamander genes that help regrow limbs, using gene therapy to restore digit growth in mice.