GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

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

Nanoscope Therapeutics has received Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations for ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Medicaid is doing a novel payment system for the new, promising and expensive sickle cell treatment. It may become a model ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Neurodegenerative diseases (NDDs) represent a profound challenge to global health, characterized by progressive neuronal dysfunction and loss, leading to ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source([1]) . The FDA approved three ...

Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting friends. It was a contrast to how Cole, 18, spent part of the 2024 holiday ...

Fred Hutchinson researchers are testing a simple idea with surprisingly big potential: a special type of foam that makes gene therapy more efficient.