Aptamer Group has announced a new partnership with BaseCure Therapeutics for the development of optimer-targeted gene therapies. The ability to precisely target gene therapies, like siRNA-based therapeutics, to specific cells and tissues using optimers offers exciting new opportunities for the field of gene therapy.
BaseCure Therapeutics is a pre-clinical stage biotech company dedicated to the discovery and development of innovative siRNA-based medicines. Aptamer is working with BaseCure Therapeutics to identify optimer binders that identify specific cell types that might be developed as potential delivery vehicles for siRNA uptake into target cells and tissues.
siRNA therapeutics form the largest portion of the rapidly developing gene therapy market, demonstrating excellent potential in the clinic. 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. This new partnership between Aptamer and BaseCure Therapeutics aims to support the future development of more targeted gene therapies.
Optimer binders are oligonucleotide affinity ligands that can be used as antibody alternatives. 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. Optimer binders can be combined with various payloads, from small molecule drugs to gene therapies, such as BaseCure Therapeutics’ siRNA. The ability to achieve optimer-directed targeted delivery would offer exciting new therapeutic opportunities of siRNA-mediated gene knockdown in these target cells and tissues.
Dr Arron Tolley, Aptamer's CEO, commented: “We’re excited to work with BaseCure Therapeutics to develop optimer delivery vehicles that improve the effectiveness of their siRNA therapies. Targeted delivery remains a significant challenge to the therapeutic oligonucleotide market, so we are pleased to be able to offer solutions for this using our optimer platform. I look forward to updating the market regarding progress in due course.”