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CTX310

Phase 1

Cardiovascular | Small molecule | Metabolic |CRISPR Therapeutics AG|Last Updated: May 22, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment90
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07491172A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory DyslipidemiasPHASE1 RECRUITING 90Jun 21, 2024Jun 1, 2028May 22, 202618 United States, Australia +2
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Study Endpoints
Primary Endpoints
To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments
From CTX310 infusion up to 12 months

Incidence of dose-limiting toxicities and frequency of adverse events

Secondary Endpoints
To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Over 12 months, compared to baseline
To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
From CTX310 infusion up to 12 months
To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
From CTX310 infusion up to 12 months
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CTX310EXPERIMENTALSubjects will receive an intravenous (IV) infusion.
Interventions
NameTypeDescription
CTX310DRUGCTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3).
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites18

Key Inclusion Criteria: 1. Age of ≥18 and ≤75 years at the time of signing the informed consent. 2. Able to provide written informed consent. 3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL i...

Countries:United StatesAustraliaNew ZealandUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07491172primaryCompletionDate: changed
LOWMay 24, 2026NCT07491172studyFirstPostDate: changed