Prenatal gene therapy via mRNA LNPs fixes faulty genes in fetal brain cells in animal model of neurodevelopmental syndrome.

A single treatment with, a CRISPR-Cas9 based gene editing therapy, is enough to replace the daily medication of patients with ...

Some prior research had shown that gene therapy would not lower the amount of time children spend on ventilator support.

Medera Inc. today announced that its clinical-stage gene therapy subsidiary, Sardocor Corp., has successfully completed its ...

Intellia has reported positive results from its ongoing Phase I/II trial of NTLA-2002, a CRISPR-based gene editing therapy ...

GenSight Biologics is weeks away from running out of money. Again. | GenSight Biologics is weeks away from running out of ...

CHILDREN in Spain who have been deaf from birth are hearing for the first time after taking part in gene therapy trials. Four ...

Acquisition creates a leading, clinical-stage company focused on the development of gene therapy treatments for rare inherited retinal degenerations New OPGx-LCA5 Phase 1/2 6-month data demonstrate sa ...

Dyno Therapeutics has formed a new research partnership with Roche, focusing on the development of next-generation ...

Positive results for Intellia Therapeutics, Inc.'s NTLA-2002 in treating HAE patients. Stock price dips, but the program ...

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The CRISPR specialist said that a single 50mg infusion of NTLA-2002 – which switches off a gene involved in the potentially ...