Aptamer Group Identifies Novel Target Enabling Liver Fibrosis Gene Therapeutics

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Using its proprietary Optimer delivery vehicle, the company said it uncovered a previously unexploited protein marker expressed on activated hepatic stellate cells.

Hepatologist holding an anatomical model of the liver. Concept of liver health, prevention, liver function, medical consultation, liver diseases, and clinical monitoring. Isolated. | Image Credit: © Tom - stock.adobe.com

Hepatologist holding an anatomical model of the liver. Concept of liver health, prevention, liver function, medical consultation, liver diseases, and clinical monitoring. Isolated. | Image Credit: © Tom - stock.adobe.com

Aptamer Group, a next-generation synthetic binder developer headquartered in York, United Kingdom, has announced its identification and preclinical validation of a novel molecular target on hepatic stellate cells (HSCs), which the company said it discovered by leveraging a delivery vehicle based on Aptamer’s proprietary Optimer technology (1). As one of Aptamer’s current goals is cell-specific drug delivery approaches, the company said its discovery significantly advances the application of targeted delivery systems for fibrotic disease.

Potential breakthrough in fibrosis therapy

Key Takeaways

  • Aptamer Group has identified and validated a novel cell surface target on hepatic stellate cells, advancing precision delivery for fibrotic liver diseases.
  • The Optimer platform enables cell-specific gene silencing via phenotype-specific markers, potentially overcoming key barriers in RNA-based fibrosis therapies.
  • As fibrosis therapies gain traction, the global market is projected to surpass $20 billion by 2030, with recent regulatory progress seen for drugs like CNP-104 and Livdelzi.

The pathological process of fibrosis, Aptamer said, is common and yet has no effective, curative treatments (1). Progression of fibrosis, which underlies cirrhosis of the liver, hepatocellular carcinoma, and other chronic liver diseases, is driven mainly by HSCs activating and proliferating, thereby secreting extracellular matrix components that form fibrotic scars.

Aptamer’s discovery workflows are designed to isolate high-affinity binders against cell-specific epitopes (1). Target confirmation was performed through both proteomic and transcriptomic profiling, the company said, as well as other studies and assays in models of liver fibrosis cells. In contrast to traditional ligand-receptor targeting, Aptamer said its approach focuses on phenotype-specific markers that are unique to fibrotic pathology—leading to greater precision in the process of delivery.

Aptamer said its platform is compatible with a variety of oligonucleotide-based payloads.

“Targeting hepatic stellate cells with high precision has been a major bottleneck in translating RNA-based therapies to fibrotic liver diseases,” said Arron Tolley, PhD, chief executive officer of Aptamer Group, in a press release (1). “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.”

Other treatments on an expanding market

According to Aptamer Group, the global fibrosis therapeutics market is projected to exceed $20 billion by 2030 (1). Numerous treatments for liver diseases are in development or already clearing regulatory hurdles.

One such therapy is Cour Pharmaceuticals’ CNP-104, which was granted orphan drug designation by FDA in January 2025 for treatment of primary biliary cholangitis (PBC) (2). CNP-104 is a biodegradable nanoparticle that encapsulates the E2 component of the mitochondrial pyruvate dehydrogenase complex, which is a key autoantigen in PBC, an autoimmune liver disease that can lead to fibrosis, cirrhosis, and ultimately liver failure.

In December 2024, the European Medicines Agency’s Committee for Medicinal Products for Human Use recommended an orphan drug approval for another treatment for PBC, Gilead Sciences’ Livdelzi (seladelpar) (3). FDA granted this treatment an accelerated approval in August 2024 (4).

References

1. Aptamer Group. Aptamer Group Identifies Novel Target to Enable Cell-Specific Gene Therapy for Liver Fibrosis. Press Release. June 30, 2025.
2. Cour Pharmaceuticals. Cour Pharmaceuticals Secures FDA Orphan Drug Designation for CNP-104 in Primary Biliary Cholangitis. Press Release. Jan. 8, 2025.
3. Gilead Sciences. Gilead’s Seladelpar Receives Positive CHMP Opinion for Primary Biliary Cholangitis. Press Release. Dec. 13, 2024.
4. Gilead Sciences. Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by US FDA. Press Release. Aug. 14, 2024.

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