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

Yes! I want to get the latest chemistry news from C&EN in my inbox every week. ACS values your privacy. By submitting your information, you are gaining access to C&EN and subscribing to our weekly ...

University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

CRISPR gene editing based treatment reduced HAE attacks rates by up to 80%. NTLA-2002 was well tolerated by patients. The ...

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

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

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

Phase 1 study and Investigator Initiated Trial evaluating KL003 for patients with beta thalassemia demonstrated an encouraging efficacy signal ...

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

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