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 ...

Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing ...

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

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

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 ...

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 ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. The Food and Drug Administration on Monday approved a ...

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

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

A new chapter is unfolding for gene therapies in Europe. With retinal programs advancing into Phase I/II trials and ...

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 ...