Victorian Heart Hospital Leads Global Trial
Commencing on 21st May, the Victorian Heart Hospital will lead the first-in-human clinical trial of a cholesterol-lowering genetic therapy. Scribe Therapeutics developed the investigational therapy STX-1150, marking the first global test at this site.
By targeting the PCSK9 gene in the liver, STX-1150 seeks to reduce LDL cholesterol, a leading cause of cardiovascular disease. The therapy utilises a CRISPR-based approach known as epigenetic silencing, providing long-term cholesterol reduction with a single infusion without permanently altering DNA.
According to Professor Stephen Nicholls, Director of the Victorian Heart Hospital, “The best way to treat heart disease, the leading cause of death globally, is to prevent it.” He emphasised the potential of STX-1150 in transforming cardiovascular risk management.
Trial Details and Participants
Evaluating the safety, tolerability, and biological effects of STX-1150, the Phase 1 study will involve adults with elevated LDL cholesterol at increased cardiovascular risk. Up to 64 participants from Australia and New Zealand will be monitored for one year after treatment.
After gaining regulatory approval from Australia’s Therapeutic Goods Administration, the trial explores genetic approaches for preventing cardiovascular disease. The Victorian Heart Institute plays an essential role in these research efforts.
Professor Nicholls expressed, “This new investigational therapy is designed to provide sustained cholesterol reduction following a single treatment, representing the new frontier of cardiovascular medicine.”
In partnership with Monash University, the Victorian Heart Hospital stands as Australia’s first dedicated cardiac hospital, advancing heart health through cutting-edge research and clinical care. Its involvement in this trial highlights its commitment to pioneering innovative treatments.
STX-1150’s novel approach, using CRISPR-based epigenetic silencing, has attracted international interest. This innovation might lead to more effective management of cardiovascular diseases worldwide.
As the trial progresses, researchers aim to collect significant data on the long-term effects of STX-1150, potentially leading to broader genetic therapies in cardiovascular treatment. The participation of Australian and New Zealand individuals underscores the region’s leading role in medical research.
Last updated: 22 May 2026, 4:50 pm
