Prime Medicine Showcases Preclinical Advances in Wilson’s Disease Program at ESGCT Congress

Prime Medicine, Inc., a biotechnology company specializing in innovative genetic therapies, recently revealed promising data from preclinical studies using a proprietary, universal lipid nanoparticle (LNP) platform. This platform is designed to deliver Prime Editors, which are gene-editing tools, to the liver to correct disease-causing mutations. The results, presented at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, showcased successful correction of genetic mutations linked to Wilson’s Disease and Glycogen Storage Disease Type 1b (GSD1b) in animal models.

Prime Medicine's research demonstrated that their LNP platform, which targets liver cells using a GalNAc-ligand, achieved significant gene editing efficiency. In studies involving both mice and non-human primates (NHPs), the platform successfully corrected mutations without causing any detectable off-target edits or safety concerns. Specifically, for Wilson’s Disease, a rare condition characterized by copper accumulation in the liver and brain, the LNP platform corrected up to 80% of the H1069Q mutation in mice and achieved precise editing in NHPs, significantly reducing copper levels in the liver.

Wilson’s Disease, if untreated, can lead to severe liver damage and neurological decline. The disease is caused by mutations in the ATP7B gene, which is responsible for copper metabolism. The Prime Editors developed by Prime Medicine target the two most common mutations in this gene, H1069Q and R778L, aiming to restore normal copper processing in the liver. Research indicates that correcting these mutations in just 20-30% of liver cells could potentially cure the disease.

Prime Medicine's Chief Scientific Officer, Dr. Jeremy Duffield, highlighted the significance of these findings. He emphasized that the liver-targeted LNP platform represents a major advancement in gene editing technology, offering a reusable and precise method to address various liver diseases. The modular nature of the platform allows for the introduction of different guide RNAs to target diverse genetic mutations, which enhances its therapeutic potential.

The company is also working on Glycogen Storage Disease Type 1b (GSD1b), a condition that disrupts glucose metabolism. Preclinical data showed that the LNP platform restored normal glycogen metabolism in a humanized mouse model and achieved high levels of precise liver cell editing in NHPs. This success supports the platform’s versatility and potential application in other liver-related diseases.

Dr. Duffield announced that Prime Medicine plans to initiate IND-enabling activities for their Wilson’s Disease program by the end of 2024, with the goal of filing an Investigational New Drug (IND) application in the first half of 2026. This timeline marks a significant step towards bringing these novel therapies to clinical trials and, eventually, to patients in need.

Prime Medicine's approach utilizes Prime Editing technology, which is designed to make precise genetic corrections with minimal unintended modifications. This technology offers the potential to address a wide range of genetic mutations and is adaptable to various tissues and cell types. The company's diversified portfolio includes programs in hematology, immunology, oncology, liver, and lung diseases. Each program targets diseases with well-understood biology and clear clinical pathways, providing a foundation for future expansion into additional therapeutic areas.

Overall, Prime Medicine aims to leverage the broad capabilities of Prime Editing to develop curative genetic therapies across a spectrum of diseases. Their ongoing research and development efforts hold promise for transformative treatments that could impact millions of patients worldwide.