A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If successful, it could be the first approved genetic-silencing method on the market, ...

For the first time, the tools to rewrite human biology are moving from speculative fiction into regulated clinics and ...

Advancements in AI, multi-omics, and genome editing are transforming life sciences and biotechnology, addressing new ...

Researchers at the Jackson Laboratory (JAX), the Broad Institute of MIT and Harvard, and Yale University, have used artificial intelligence (AI) to design thousands of new DNA switches that can ...

Single cell gene expression studies often rely on single-cell RNA sequencing (scRNA-seq) alone. While scRNA-seq reveals which genes are turned on, it does not capture protein expression or changes to ...

Krystal Biotech reports positive clinical data for KB408 and KB407, demonstrating safety and efficacy in AATD and cystic fibrosis patients. Krystal Biotech, Inc. has announced promising clinical ...

Earlier this week, Krystal Biotech reported positive interim data from the highest-dose cohort of its Phase 1 CORAL-1 trial, ...

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.