Aptamer Group Identifies Novel Target to Enable Cell-Specific Gene Therapy for Liver Fibrosis

Aptamer unveils novel biomarker for targeted liver fibrosis therapy

Optimer breakthrough targets liver scarring cells, positioning Aptamer in $20bn fibrosis market

Aptamer Group plc today announced the identification and preclinical validation of a novel molecular target on hepatic stellate cells (HSCs), enabling targeted delivery of gene therapeutics for liver fibrosis using its Optimer® delivery vehicle. The findings represent a significant advance in the application of targeted delivery systems for fibrotic disease and support Aptamer’s development of cell-selective drug delivery approaches.

Fibrosis, a common pathological process underlying liver cirrhosis, hepatocellular carcinoma, and other chronic liver diseases, remains without effective curative treatments. The progression of fibrosis is largely driven by the activation and proliferation of HSCs, which secrete extracellular matrix components that form the fibrotic scar. Selective delivery of therapeutics to these cells remains a major challenge in the field.

Leveraging its proprietary Optimer technology, Aptamer Group has identified a previously unexploited protein marker expressed on activated HSCs. The team developed an Optimer® delivery vehicle that binds this target with high specificity, enabling direct delivery of RNA therapies to fibrogenic cells. When conjugated to siRNA the Optimer® delivery vehicle suppresses pro-fibrotic signalling pathways, resulting in reduced markers of fibrosis and decreased HSC migration and proliferation in preclinical liver fibrosis models.

“Targeting hepatic stellate cells with high precision has been a major bottleneck in translating RNA-based therapies to fibrotic liver diseases,” said Dr Arron Tolley, Chief Executive Officer of Aptamer Group. “By isolating a disease-relevant cell surface target and validating Optimer®-mediated delivery, we’ve demonstrated a viable route to cell-specific gene silencing. This positions the platform as a promising component of future fibrosis therapeutics.”

The Optimer® binders were developed using Aptamer’s proprietary discovery workflows, designed to isolate high-affinity binders against cell-specific epitopes, even in complex tissue contexts. Characterization of the Optimer® delivery vehicle included target confirmation through proteomic and transcriptomic profiling, as well as binding studies and functional assays in liver fibrosis cell models.

This approach differs from traditional ligand-receptor targeting by focusing on phenotype-specific markers unique to fibrotic pathology, allowing greater precision in therapeutic delivery. In vitro studies have demonstrated that Optimer® conjugates can selectively target hepatic stellate cells in fibrotic liver tissue models without significant off-target uptake in healthy hepatocytes.

The company is now preparing for in vivo validation studies through outsourced collaborations, aiming to establish the performance of the Optimer®-siRNA system in animal models of liver fibrosis. Concurrent discussions with pharmaceutical partners are ongoing to explore licensing and co-development opportunities.

The fibrosis therapeutics market is projected to exceed $20 billion globally by 2030, with an increasing focus on targeted RNA-based strategies. Aptamer’s platform, which is compatible with a wide range of oligonucleotide-based payloads, including siRNA and antisense oligos, offers a modular solution for addressing fibrosis and other fibroproliferative disorders.

For further information, please contact:  

Aptamer Group plc

Dr Jane McLeod

+44 (0) 7930683434

About Aptamer Group

Aptamer Group is a leading developer of next-generation synthetic binders delivering innovation to the life sciences industry. The Group develops Optimer® binders, advanced molecules that work like antibodies by attaching to specific targets in the body. These binders are used in medicine, diagnostic tests, and research tools, offering benefits like high stability, reliable performance, and lower costs compared to traditional antibodies.

Aptamer operates a fee-for-service business in the $210 billion market for antibody alternatives, working with all top 10 global pharmaceutical companies. It is also building valuable Optimer® assets with partners, aiming for future licensing revenue.