STX-1150 is an investigational therapy designed to lower LDL-C by silencing a gene called PCSK9 in the liver. STX-1150 does not edit or permanently change the gene. STX-1150 comprises an mRNA and guide RNA (gRNA) delivered via lipid nanoparticles (LNP) for intravenous infusion. The mRNA produces a protein that switches off the PCSK9 gene expression without altering the DNA sequence. This process leverages natural mechanisms that regulate gene activity.
The study will enroll up to 64 participants with elevated LDL-C across sites in Australia and New Zealand. The follow-up period will be up to 1- year post-treatment.
Elevated serum LDL-C with or without LDL-C lowering medication
Willing and able to give informed consent before initiation of any study-related procedures and willing to comply with all required study procedures
Patients with history of an ASCVD event </= 6 months.
Any uncontrolled or serious disease, or any medical or surgical condition that may interfere with participation
Diagnosis of familial hypercholesterolemia
Active or history of liver disease
Single group
STX-1150
DrugDrug: STX-1150 is an investigational product designed to epigenetically silence the PCSK9 gene. Epigenome modulation offers a way to silence genes without changing their underlying DNA sequence.
Incidence and Severity of Treatment-Emergent Adverse Events (TEAEs)
Incidence and Severity of Serious Adverse Events (SAEs)
Incidence and Severity of Adverse Events of Special Interest (AESI)
Incidence of dose-limiting toxicities (DLTs)
Percent Change from Baseline in Plasma PCSK9 Concentration
Percent Change from Baseline in LDL-C
Plasma Concentrations of STX-1150 Lipid Components
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Monash University
Lead sponsor
Scribe Therapeutics Inc.
Collaborator
