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A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias
Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.
Generated
Jun 25, 2026
Report code
NCT07491172-Jun 25, 2026
NCT ID
NCT07491172
Status
RECRUITING
Phase
Phase 1
Sponsor
CRISPR Therapeutics AG
Executive brief
Investment-Ready Snapshot
CRISPR Therapeutics AG is advancing CTX310, a novel lipid nanoparticle formulation of CRISPR-Guide RNA-Cas9 nuclease, targeting refractory dyslipidemias. This Phase 1 trial aims to establish safety and tolerability, addressing a significant unmet need in patients with persistent dyslipidemias unresponsive to existing therapies. The global dyslipidemia treatment market is substantial, with increasing prevalence driving demand for innovative therapies. Successful outcomes could position CTX310 as a first-in-class treatment, enhancing CRISPR Therapeutics' competitive edge in the gene editing space and potentially leading to lucrative partnerships or acquisitions. Diligence should focus on regulatory pathways and reimbursement strategies, given the complexities surrounding gene editing technologies.
Source & freshness
Provenance
https://clinicaltrials.gov/study/NCT07491172
Indication
Cardiovascular
Modality
protein therapy
Target
Angiopoietin-like 3 (ANGPTL3) gene
Intervention
CTX310
Source record
Protocol Description
Detailed source ingestion pending.
Source record
Outcome Measures
Detailed source ingestion pending.
Source record
Eligibility
Detailed source ingestion pending.
AI analysis
Known Results And Readout Context
Detailed source ingestion pending.
IP intelligence
Patent And IP Landscape
Detailed source ingestion pending.
Source record
Contacts
Detailed source ingestion pending.