With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...

The Barbara Ann Karmanos Cancer Institute in Detroit has become the first and only independent cancer center in the U.S. to ...

Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

Hemgenix for hemophilia B is temporarily unavailable, says maker CSL Behring ...

Sarepta Therapeutics is still assessing how best to prevent liver injury from its commercialized Duchenne muscular dystrophy gene therapy. But Sarepta partner Hansa Biopharma has encouraging ...

AUSTIN, Texas, Oct. 28, 2025 /PRNewswire/ -- Genprex, Inc. (GNPX) ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for ...

A genetic variant carried by at least one-fifth of the population may do far more than raise the odds of developing Alzheimer ...

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene therapy for severe hemophilia A.

Mayo Clinic researchers used milk-based nanoparticles to deliver siRNA into bile duct cancer cells. This targeted therapy ...

In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...

KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week. KJ has spent the majority of his ...