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 ...

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 ...

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 ...

Favorable early safety and initial efficacy data from BEST1 program highlighted at premier gathering of global retinal experts with additional ...

Scientists have identified a crucial molecular switch that decides whether pancreatic cancer cells resist chemotherapy or respond to it. The key player, a gene called GATA6, keeps tumours in a more ...

Cure Rare Disease (CRD), a non-profit biotechnology organization, today announced a landmark partnership with the LGMD2L Foundation. This collaboration is backed by a generous commitment of $7.65 ...