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CTX340

Phase 1

Hypertension | Small molecule | Cardiovascular |CRISPR Therapeutics AG|Last Updated: Aug 11, 2026

Target and mechanism

Molecular targetAGT
Target classGene
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment69

FDA Designations

No designations recorded

Clinical trial landscape

CTX340 · 1 trial · 1 indication

Phase 1 1
NCT07758634A Trial Evaluating Safety, Tolerability and Efficacy of CTX340 in Participants With HypertensionHypertension
RECRUITING69 Analytics
PHASE1RECRUITING
A Trial Evaluating Safety, Tolerability and Efficacy of CTX340 in Participants With Hypertension
HypertensionUnlock trial analytics

Study Endpoints

Primary Endpoints

Phase 1: Incidence of dose-limiting toxicities (DLTs)
Up to 12 months

To evaluate the safety and tolerability of a single ascending dose of CTX340 in participants with hypertension to determine the recommended Phase 2 dose (RP2D).

Phase 2: Percentage change in circulating angiotensinogen (AGT) concentration from baseline
Through 6 months of follow-up.

To evaluate the pharmacodynamics (PD) effect of CTX340 at the recommended Phase 2 dose (RP2D)

Secondary Endpoints

To assess the safety of CTX340
From CTX340 infusion up to 12 months
To assess the effect of CTX340 on systolic blood pressure (SBP) by ambulatory blood pressure monitoring (ABPM)
From CTX340 infusion up to 12 months
To assess the pharmacodynamics (PD) effect of CTX340
Over 12 months, compared to baseline
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Phase 1: CTX340EXPERIMENTALPhase 1 is An Open-Label, Ascending Dose Design.
Phase 2: Recommended Phase 2 Dose CTX340EXPERIMENTALPhase 2: Recommended Phase 2 Dose CTX340.
Phase 2: PlaceboEXPERIMENTALPhase 2: Placebo.

Interventions

NameTypeDescription
CTX340DRUGCTX340 is an in vivo gene editing therapy designed to utilize clustered regularly interspaced short palindromic repeats-CRISPR-associated protein 9 (CRISPR-Cas9) to target and disrupt human angiotensinogen (AGT) gene in liver.
PlaceboDRUGOne of the arms in Phase 2 will be placebo.
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites2

Key Inclusion Criteria: 1. Age: ≥18 and ≤75 years. 2. Body mass index ≤40 kg/m2. 3. 24-hour mean ambulatory blood pressure monitoring (ABPM) systolic blood pressure (SBP) measurement of ≥130 mm Hg but ≤160 mm Hg despite treatment 4. Be on treatment with ≥4 antihypertensive therapies at effective do...

Countries:United StatesAustralia
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Recent Changes (Last 90 Days)

LOWAug 11, 2026NCT07758634NEW_TRIAL: changed
LOWAug 11, 2026NCT07758634NEW_TRIAL: changed

Frequently asked questions about CTX340

What is CTX340 used for?

CTX340 is an investigational small molecule being developed for the treatment of hypertension, a cardiovascular condition characterized by chronically elevated blood pressure. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

What does CTX340 target?

CTX340 targets the AGT gene, which encodes angiotensinogen, a key precursor in the renin-angiotensin system that regulates blood pressure. By targeting this gene, CTX340 aims to modulate the production of angiotensinogen to potentially lower blood pressure in patients with hypertension.

Who makes CTX340?

CTX340 is being developed by CRISPR Therapeutics AG, a biopharmaceutical company publicly traded under the ticker symbol CRSP. The company is conducting clinical trials to evaluate the safety, tolerability, and efficacy of CTX340 in participants with hypertension.

What phase is CTX340 in?

CTX340 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory agencies and is still undergoing clinical trials to assess its safety and efficacy in humans.

What clinical trials is CTX340 in?

CTX340 is being evaluated in a Phase 1 clinical trial with the identifier NCT07758634. This trial is recruiting participants with hypertension in the United States and Australia, with a planned enrollment of 69 participants aged 18 years and older.