CRSP-CVD-402
Research type
Research Study
Full title
A Phase 1/2 Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety, Tolerability, and Efficacy of a Lipid Nanoparticle Formulation of CRISPR–Guide RNA–Cas9 Nuclease (CTX340) for In Vivo Editing of the Angiotensinogen (AGT) Gene in Participants with Hypertension
IRAS ID
1013786
Contact name
Madhuri Agarwal
Contact email
Sponsor organisation
CRISPR Therapeutics AG
Clinicaltrials.gov Identifier
Research summary
Hypertension (high blood pressure) is a major risk factor for diseases of the heart and blood vessels. There are approved medicines for the treatment of hypertension, but these are not always effective in everyone. When approved medicines fail to control high blood pressures to safe levels, this is called uncontrolled hypertension.
This research study is being conducted to study how a potential new medicine (called CTX340) may be useful in treating uncontrolled hypertension. The research will look at how safe, tolerable, and effective CTX340 is. CTX340 is designed to work in the liver and reduce blood pressure within the body by partially turning off of genes related to hypertension. Participants in this study will receive either one or two doses of CXT340 via an intravenous infusion (injection into a vein).
CTX340 is considered an investigational drug. This means that it has not yet been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) for regular use. However, it can be used in this type of research study (known as a clinical trial).
Approximately 69 participants with uncontrolled hypertension will take part in this clinical trial. These participants will attend regular visits at their medical centre and their health will be monitored. In addition to the medical centres in the UK, this clinical trial is also planned to take place in additional countries around the world.CRISPR Therapeutics AG is the “Sponsor” of this research study and is responsible for the development and oversight of the study and CTX340.
REC name
North East - York Research Ethics Committee
REC reference
26/NE/0077
Date of REC Opinion
28 May 2026
REC opinion
Further Information Favourable Opinion