First-in-human trial of cholesterol-lowering genetic therapy to begin at Victorian Heart Hospital
Monash University and Monash Health
The Victorian Heart Hospital and the Victorian Heart Institute will lead a first-in-human clinical trial of a new investigational genetic therapy that aims to lower cholesterol in people at increased risk of cardiovascular disease.
The Victorian Heart Hospital, operated by Monash Health in partnership with Monash University, will be the first clinical trial site globally to begin testing the investigational therapy STX-1150, developed by Scribe Therapeutics.
The therapy is designed to reduce LDL (‘bad’) cholesterol by targeting a gene in the liver called PCSK9, a well-established regulator of cholesterol levels and cardiovascular risk. Elevated LDL cholesterol is a major cause of atherosclerotic cardiovascular disease, including heart attacks and strokes.
STX-1150 uses a next-generation CRISPR-based approach known as epigenetic silencing. Delivered as a one-time infusion, the therapy is designed to reduce cholesterol levels for an extended period without permanently altering a person’s DNA.
Principal Investigator of the study Professor Stephen Nicholls, Director of the Victorian Heart Hospital and Victorian Heart Institute, and Professor of Cardiology at Monash University, said the trial represented another major step forward in developing more durable approaches to cardiovascular prevention.
‘The best way to treat heart disease, the leading cause of death globally, is to prevent it,’ Professor Nicholls said.
‘While existing cholesterol-lowering therapies are highly effective, many people still struggle to maintain long-term treatment due to cost, access, side effects, or the burden of ongoing medication.’
‘This new investigational therapy is designed to provide sustained cholesterol reduction following a single treatment, which could significantly change how we manage cardiovascular risk in the future. This represents the new frontier of cardiovascular medicine.’
‘It is incredibly exciting that Victorians and Australians will again play a leading role in the development of next-generation genetic therapies for heart disease.’
The Phase 1 study will assess the safety, tolerability and biological effects of STX-1150 in adults with elevated LDL cholesterol who are at increased cardiovascular risk.
The trial plans to enrol up to 64 participants across sites in Australia and New Zealand, with participants monitored for one year following treatment.
The study follows regulatory clearance from Australia’s Therapeutic Goods Administration and builds on growing international interest in genetic approaches to cardiovascular disease prevention.
About the study
- The Phase 1 trial will evaluate STX-1150 in adults with elevated LDL cholesterol and increased cardiovascular risk.
- The study is designed as an open-label, single ascending dose trial followed by a dose expansion phase.
- Up to 64 participants are planned to be enrolled across Australia and New Zealand.
- The Victorian Heart Hospital will serve as the initial clinical trial site.
- Participants will be followed for one year after treatment.
About the Victorian Heart Hospital
The Victorian Heart Hospital, operated by Monash Health in partnership with Monash University, is Australia’s first dedicated cardiac hospital, bringing together world-leading clinical care, research and education to advance heart health and improve outcomes for patients across Victoria and beyond.
About the Victorian Heart Institute
Monash University’s Victorian Heart Institute is a health and medical research organisation based in the Victorian Heart Hospital. It unites multi-disciplinary experts to deliver excellence and innovation in cardiovascular research, education and training.
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