Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

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

Abu Dhabi achieved a medical milestone on January 5, 2026, with the UAE's first gene-therapy injection for inherited blood disorders. CASGEVY, utilizing CRISPR-Cas9, corrects faulty DNA in conditions ...

The U.S. Food and Drug Administration (FDA) on Monday extended its review timeline for REGENXBIO Inc.’s (NASDAQ:RGNX) Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for ...