Aptamer announces new data at ASGCT

Aptamer Group plc, the leading developer of next-generation synthetic binders delivering innovation to the life science industry, presented new data on its Optimer therapeutic delivery vehicle for liver fibrosis at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in New Orleans, LA (13-17 May 2025).

Developed through an internal research program and in collaboration with AstraZeneca, the Optimer system is based on next-generation synthetic oligonucleotide ligands designed to achieve tissue-specific targeting. The latest findings show that the Optimer construct selectively targets fibrotic tissue across multiple organ systems—including liver, kidney, skin, lung, and heart—and is compatible with both mouse and rat models, for convenient transition to preclinical animal studies.

These results establish the translational potential of the Optimer platform, which has shown compatibility with small interfering RNAs (siRNAs) and demonstrated effective delivery of three distinct siRNA payloads to fibrotic sites. This modularity supports the platform’s adaptability for a broad range of therapeutic applications in the context of fibrosis, where there is high unmet need.

The platform's ability to engage multiple fibrotic tissues suggests a shared pathophysiological targeting mechanism that may be exploited to develop precision anti-fibrotic therapies. These findings position the Optimer platform as a promising candidate for further preclinical validation and potentially clinical development, especially given the high unmet need and limited treatment options in fibrotic disease.

These results broaden the scope of Aptamer’s internal development program, indicating our delivery vehicle could address a broad range of disease areas. While fibrotic liver disease remains a major focus due to its large market size and limited treatment options, the potential to expand into pulmonary, renal, cardiac, and dermal fibrosis greatly increases the scope of this delivery system.

With the market for liver fibrosis treatments set to grow at a compound annual growth rate of 24% through 2035, driven by new drugs under development and rising disease prevalence, the Group is well-positioned to capitalize on this momentum. Aptamer’s ongoing collaboration with AstraZeneca continues to generate compelling data, and multiple pharmaceutical companies have initiated discussions to explore partnerships over the next few months.”

Dr Arron Tolley, Chief Executive Officer, Aptamer Group