A single dose of the CRISPR-based gene-editing therapy NTLA-2002 reduced angioedema attacks and led to sustained reduction in ...

The incubator is designed to draw cell and gene therapy researchers and drugmakers to work at the hub, where they can develop ...

After 44 days, Kendric Cromer, 12, left the hospital. While his family feels fortunate that he was the first to receive a ...

CHICAGO -- A mutation-agnostic gene therapy for retinitis pigmentosa (RP) showed promise for improving vision in severely ...

A high dose of the investigational gene therapy AAV-GAD, which delivers the glutamic acid decarboxylase ( GAD) gene to the ...

Gov. Kathy Hochul on Tuesday announced a $150 million investment in a new state-of-the-art cell and gene therapy hub to be ...

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

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

Eye drug maker Ocuphire Pharma is acquiring gene therapy developer Opus Genetics in an all-stock transaction that will see ...

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

With an upfront payment of $50 million from Roche, the partnership will leverage Dyno Therapeutics’ in vivo gene therapy ...

The regulator agreed to allow Sangamo Therapeutics to use data to seek accelerated approval for its Fabry gene therapy ...