GHP Q2 2024

26 GHP Q2 2024 t can be difficult to get an active pharmaceutical ingredient to where it is needed inside the body while ensuring it remains intact. Many molecules, including RNA, decay rapidly and often do not reach their target destination. If these molecules are to be transformed into effective therapeutics, they require a robust delivery system. Procavea Biotech have developed a unique protective shell that can package and protect RNA molecules. The technology is inspired by Nature’s solution to the problem of delivering RNA molecules into cells: viruses. However, the system is free from any virus-derived components. Procavea’s delivery system is a nanoscale cage composed of 24 identical proteins that can accommodate smaller RNA molecules, known as small interfering RNA (siRNA). The company is currently focused on the Antisense and siRNA therapeutics market, providing a solution to expand the development of siRNA therapeutics beyond diseases affecting the liver. Tom shares, “RNA-based therapeutics have shown great potential in treating a wide range of diseases due to their ability to target almost any disease-causing gene within a cell. However, the breadth and complexity of human disease demands innovative approaches beyond existing technologies to provide continued benefits to patients. “To address this need, we created a unique, nonviral oligonucleotide delivery system from an artificial protein cage, called OP. Protein nanostructures offer uniform size, a defined and atomically resolved structure, broad chemical functionality, and inherent biodegradability. Additionally, recombinant protein production is simple, scalable, economical, and allows both genetic and chemical modification to optimise pharmacokinetics and target particles to specific tissues.” Procavea Biotech’s technology platform uses an artificial, computationally designed protein structure that is highly stable, allowing it to be extensively engineered for specific therapeutic applications. Its porous structure enables fast and quantitative in vitro loading with diverse biological or synthetic cargo molecules. The OP system is non-toxic and able to enter various cell types to deliver its cargo. The technology has been developed at ETH Zurich and is protected by two patents. Apr24344 I Based in Switzerland, Procavea Biotech is dedicated to revolutionising the delivery of RNA therapeutics. With its protein-based RNA delivery platform, the company strives to solve the problem of extrahepatic delivery to realise the untapped potential of RNA medicines. We speak to Procavea Biotech’s founder, Tom Edwardson, to find out more about the company’s pioneering work. Tom Edwardson says, “Our technology is clearly differentiated from currently available siRNA delivery solutions. The economic, scalable production, high stability, and a quantitative one-step formulation with the active pharmaceutical ingredient are key selling points. Exploiting the engineerability to target the particles to different organs and tissue types opens solutions to the unmet needs in oligonucleotide delivery. We are currently focused on developing a first-in-class product in a disease area that the state-of-the-art cannot address. Specifically, by delivering oligonucleotides to an organ other than the liver.” Currently, the Procavea team is evaluating new generations of protein cages in vivo. Current delivery systems, such as lipid nanoparticles and so called GalNAc conjugates can only target the siRNA to the liver. Initial studies demonstrate that Procavea’s unique delivery solution has the clear advantage to target extrahepatic tissues and that cage variants can be engineered for different tissue distribution. Recognised in this year’s Global Excellence Awards, Procavea Biotech is dedicated to solving a major limitation in the application of RNA Therapeutics with its unique protein-based delivery technology. Tom Edwardson and his expert team anticipate a productive year of innovation on their mission to address the industry’s unmet clinical needs and revolutionise the delivery of RNA therapeutics. For its unwavering commitment to improving global health, we have bestowed on Procavea Biotech our award for RNA Therapeutics Innovator of the Year – Switzerland. Contact Details Contact: Tom Edwardson Company: Procavea Biotech Web Address: https://www.procavea.com/ The Future of Drug Delivery

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