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Aim to expedite oligonucleotide manufacturing for gene-silencing therapeutics.
July 27, 2021
By: Charlie Sternberg
CatSci Ltd, a scientifically-led, commercially-minded and award-winning innovation partner for medicines development, and Argonaute RNA, a biotech developing safe and reliable methods for transient silencing of target genes in different tissue cells, have entered a chemistry, manufacturing and control (CMC) development collaboration focused on novel therapeutic oligonucleotides. The new collaboration is underpinned with significant upfront investment into Argonaute RNA by CatSci, and the appointment of CatSci’s CEO Ross Burn as non-executive director to Argonaute RNA’s Board. The two parties will develop the CMC strategy for the production of synthetic oligonucleotides for use in Argonaute RNA’s gene-silencing platform. Argonaute RNA has an RNA interference (RNAi) platform, protected by patents both granted and pending. Its unique approach to modification of small interfering RNA (siRNA) offers a safe and effective way to temporarily silence specific gene expression and modify disease progression. A novel siRNA stabilization method enables Argonaute RNA to avoid a range of issues which have proved significant obstacles to developing siRNA drugs for use in humans. The implementation of a robust synthetic oligonucleotide CMC strategy will support the use of oligonucleotides in wider drug discovery and development programs, and further drive the expansion of CatSci’s analytical and synthetic capabilities. “The promise of oligonucleotide therapeutics is hugely exciting but there is still significant work to be done to de-risk both the discovery and the manufacturing process. Partnering with Argonaute RNA fits perfectly with our mission to become a fully integrated CMC company. Going beyond drug substance to develop our oligonucleotide capabilities will open a whole new area in which we can help accelerate medicines development,” said Dr Burn. He continued, “CatSci’s investment directly into Argonaute RNA is a signifier of our commitment to making this a truly collaborative effort, and I look forward to working closely with the team.” Anthony Parker, CEO at Argonaute RNA, commented, “We’re excited to work with CatSci in the CMC landscape and harness their expertise in solving complex drug development challenges to accelerate the development of our siRNA platform and antisense technology. Ross will be an excellent addition to our Board, and it will be great to have him as a hands-on partner as we progress the collaboration.” Dr Nigel Richardson, director of Analytical Technology & New Modalities, at CatSci, added, “Synthetic oligonucleotides are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic, but progress has been slow and many challenges remain before they can be fully integrated within medicines development. Innovative multi-disciplinary solutions are urgently needed, as well as highly specific and sensitive methods for manufacturing, quality control, and to meet regulatory standards. Our work with Argonaute RNA to develop oligonucleotides for their siRNA platform will help drive the paradigm shift needed for the manufacturing process.” Oligonucleotides can be designed to recognize any species of cellular DNA or RNA and, in theory, have the potential to modulate gene expression and affect the course of almost any disease. Once a target RNA is chosen based on its physiological significance, a complementary oligonucleotide can be synthesized, gene expression assayed, and phenotypes examined. Argonaute RNA recently secured £1.4 million ($1.9 million) in funding from a number of investors, led by The Fink Family Office with contributions from the Bristol Private Equity Club, as well as CatSci.
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