Tag: #crispr

Single IV CRISPR Dose Produces ~50% LDL and Triglyceride Reductions at One Year in Early Trial

A single IV dose of CRISPR Therapeutics’ CTX310 produced about 50% average reductions in LDL cholesterol and triglycerides at one year in a small Phase 1a trial.

$CRSP, #crispr, #lipids, #geneediting, #angptl3

Intellia’s one-time CRISPR therapy cuts hereditary angioedema attacks 87% in Phase 3; rolling FDA filing started

Phase 3 trial of Intellia's one-time in‑vivo CRISPR therapy lonvo‑z cut hereditary angioedema attacks 87%; company began rolling FDA filing.

$NTLA, #crispr, #geneediting, #biotech, #hereditaryangioedema, #intellia

Engineered miniature CRISPR achieves >80% editing in human cells and could fit inside AAV

Engineered miniature CRISPR raised editing in human cells from <10% to >80% and stays small enough to fit in AAV vectors — tests remain in cells.

#gene-editing, #crispr, #aav, #biotechnology