BaseCure Therapeutics is a pre-clinical stage biotech focused on the discovery and development of siRNA-based medicines. Aptamer is working with the biotech to identify cell targeting Optimer binders that might be developed as potential delivery vehicles for siRNA uptake into target cells and tissues.
The ability to achieve Optimer-directed targeted delivery would offer new therapeutic opportunities of siRNA-mediated gene knockdown in these target cells and tissues, according to the partners.
Addressing challenges in the siRNA therapeutics field
siRNA therapeutics form the largest portion of the rapidly developing gene therapy market, demonstrating excellent potential in the clinic, they noted.
To date, there has been FDA approval of four siRNA treatments. However, despite these successes, targeting the therapeutic to the desired cell or organ within the body and further cellular internalisation to allow its gene silencing function remains a major challenge. Hence, the focus of the new partnership: supporting the future development of more targeted gene therapies.
Optimer binders are oligonucleotide affinity ligands that can function as an antibody alternative. Selected wholly in vitro, Optimers offer the potential to tune target affinity and half-life and allow for site-directed payload conjugation for controlled Optimer: therapeutic ratios, reported Aptamer.
Moreover, the UK company said Optimer binders can be combined with various payloads, from small molecule drugs to gene therapies, such as siRNA-based therapeutics.
Viral vector deal
Aptamer also has an agreement with Bio-Works Technologies to codevelop new affinity purification resins for improved purification of gene therapy vectors.
The firm's business model is to provide contract research services on a fee-for-service basis in addition to longer-term upside potential from ongoing royalty and licensing revenues where its binders are taken forward by customers into commercial applications.
Aptamer has worked with 75% of the world’s top 20 pharma companies, as well as SMEs, large regional organisations, universities and charities.
