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

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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.

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell ...

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

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 ...

ReviR Therapeutics, a pioneering biotechnology company focused on developing novel small molecule RNA modulators for neurogenetic diseases, announced a groundbreaking research grant from the Kennedy's ...

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