Parents of 6-year-old Raiden Pham say new FDA feedback brings hope as they work to raise $2 million for future gene therapy, ...

A research team led by Albert Einstein College of Medicine scientists has developed a new strategy to engineer immune cells ...

A genetic mutation that helps animals like yaks and Tibetan antelopes survive at high altitudes may hold the key to repairing nerve damage in conditions such as cerebral paralysis and multiple ...

Lipid nanoparticles, or LNPs, are best known as the delivery system used in the COVID-19 mRNA vaccines given to billions of people worldwide. Scientists are now exploring their potential far beyond ...

Sickle cell disease—the subject of a new Seminar in The Lancet—is one of the most prevalent and fastest-growing genetic disorders worldwide. Although its true prevalence is difficult to determine ...

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

For five decades, scientists have known about a notorious cancer-causing enzyme called SRC. But they always assumed it only appeared on the inside of cells, where it sent signals that fueled tumor ...

Reported Promising Data for TN-201 and TN-401 Gene Therapies in Fourth Quarter of 2025; Additional Data Readouts and Pursuit of Regulatory ...

Ultragenyx Pharmaceutical said on Thursday its experimental gene therapy helped patients in a ​late-stage trial to better control ammonia levels linked to ‌a rare inherited disorder.

INSTALL results showed successful and safe non-viral insertion of large genetic payloads in the livers of mice when delivered by lipid nanoparticles (LNPs). In contrast, mice experienced fatal immune ...

Amerigo Scientific launched a comprehensive portfolio of GMP-grade proteins tailored to accelerate the development and ...