CityUHK develops "DNA surgery" for liver, cardiovascular diseases

City University of Hong Kong researchers are developing novel genome editing technology to treat genetic liver and cardiovascular diseases through a three-year project funded under the Hong Kong SAR Government's RAISe+ Scheme.

Led by Professor Zheng Zongli from the Department of Biomedical Sciences, the team aims to create "one-and-done" therapies using "DNA surgery" technology that directly corrects errors in patients' DNA. The project focuses on developing two core therapeutic medicines for clinical trials.

Unlike traditional gene therapies that require repeated dosing, this approach uses CRISPR genome editing with patented high-fidelity nucleases to perform durable DNA-level corrections. Engineered lipid nanoparticles deliver the editing tools precisely to target cells.

The team is preparing two drug candidates - PL-100 for a rare liver disease and PL-200 for cardiovascular disease - for investigational new drug studies and clinical trials. Preliminary pre-clinical studies in non-human primates have shown promising results with high precision and efficiency at lower dosages.

Professor Zheng stated that genome editing represents "the next frontier in medicine, not merely controlling diseases but curing them at their root," adding that Hong Kong has the potential to become a global leader in genomic medicine.