CRISPR Therapeutics starts Phase 1 trials for hypertension and AATD

CRISPR Therapeutics stated in its second-quarter business update, filed as an exhibit to a Form 8-K on 3 August 2026, that it has initiated Phase 1 clinical trials of two in vivo gene-editing candidates.

CTX340 targets angiotensinogen (AGT), the liver protein at the head of the pathway that raises blood pressure, and has received clearance of its investigational new drug application from the FDA. The trial takes patients with refractory hypertension — blood pressure that stays high despite multiple drugs. The premise is that one edit to a liver gene stands in for a pill taken daily for decades.

CTX460 targets SERPINA1 and is the first candidate produced by what the company calls its SyNTase editing platform, which the release describes as designed for precise, efficient and scalable gene correction. Its indication is alpha-1 antitrypsin deficiency, an inherited disorder in which a misfolded protein accumulates in the liver and leaves the lungs without their usual protection.

Both are single infusions rather than chronic treatment, and both are Phase 1 — the stage that asks whether a dose is tolerated, not whether it works. Neither has reported data. What changed on 3 August is that two more organ-level genetic corrections moved out of animals and into people.