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 ...
UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell ...
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
After failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells ...
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
Novartis's acquisition of Kate Therapeutics gives it a pipeline of gene therapies for three genetic neuromuscular diseases.
CRISPR gene editing based treatment reduced HAE attacks rates by up to 80%. NTLA-2002 was well tolerated by patients. The ...
Novartis has acquired Kate Therapeutics for up to $1.1 billion, the companies said today, in a deal that expands the buyer’s ...
UAB researchers have reversed metabolic dysfunction-associated steatohepatitis (MASH) in mouse models. MASH is a severe liver disease associated with obesity and type 2 diabetes that affects more than ...
Phase 1 study and Investigator Initiated Trial evaluating KL003 for patients with beta thalassemia demonstrated an encouraging efficacy signal ...
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