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.

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

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

So, we’re voting, and the outcome may end the world, or not. So, we’re voting, and just reading the news this morning put me ...

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

Ada, 9, and Lily, 12 — are the first patients on the West Coast to receive a new gene therapy offered by UCSF Benioff ...

A new study shows that a biomedical tool can successfully deliver genetic material to edit faulty genes in developing fetal ...

STAT's Readout LOUD podcast discusses Pfizer's woes, explains RFK Jr.'s 'MAHA' campaign, and mulls the latest about the Novo ...

Intellia Therapeutics' Phase 2 data shows NTLA-2002, a CRISPR gene therapy, reduced hereditary angioedema attack rates by up to 81%, with complete responses.