AFTX-201, a potential best-in-class investigational gene therapy intended to be given as a simple one-time intravenous (IV) administration, is undergoing investigational new drug (IND)-enabling ...

It could create savings that can be passed on to the consumer. High-tech startup zooms in on animal DNA to revolutionize dairy industry: 'Our mission is to make milk without a cow' first appeared ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we ...

New CRISPR tool enables more seamless gene editing — and improved disease modeling Yale scientists have developed a CRISPR technology that can assess genetic interactions on a host of immunological ...

Which Gene Therapy Technology Wins? New Data from Spherix Global Insights Reveals Emerging Hematologist Preferences in Beta Thalassemia Care Provided by GlobeNewswire Aug 14, 2025, 10:30:18 AM ...

The researchers used a gene editing approach based on CRISPR-Cas9 technology, delivered by an AAV2 vector directly into the inner ear of an adult mouse model with DFNA41.