Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...

The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA ...

For instance, Zolgensma has been approved for the treatment of spinal muscular atrophy, offering a one-time gene replacement therapy that addresses the root cause of the disease. Similarly, Luxturna ...

Novartis has acquired Kate Therapeutics for up to $1.1 billion, the companies said today, in a deal that expands the buyer’s ...

Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine ...

Novartis's acquisition of Kate Therapeutics gives it a pipeline of gene therapies for three genetic neuromuscular diseases.

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy ...

After failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells ...

CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...

The Delhi High Court sought the Centre’s response on Monday on a plea moved by a girl suffering from a rare disease, seeking ...

Epidermolysis bullosa (EB), or "butterfly disease" Affected populations: Butterfly disease is estimated to affect around 1 in ...