Defence Therapeutics has announced the publication of its new international (PCT) patent application. This demonstrates the effectiveness of Defence’s Accum™-based technology to intracellularly deliver a wide range of cargoes while avoiding endosomal entrapment.
The PCT application was published 15 June 2023 as WO/2023/102661 A1 by the World Intellectual Property Organisation (WIPO). The application contains data showing the efficient cytosolic/nuclear delivery of multiple therapeutically relevant cargoes. These include polynucleotides, recombinant proteins, and even nucleoprotein complexes.
The results contained in the PCT application demonstrate the successful nuclear delivery of functional CRISPR/Cas9. They also guide RNA ribonucleoprotein complexes by Accum™ variants.
The findings highlight the broad applicability of Defence’s Accum™-based platform technology. Furthermore, they will pave the way for additional future strategic collaborations. For example, in the fast-growing field of genome editing.
The ability of Accum™ to deliver large and functional ribonucleoprotein complexes into the nucleus of cells represents a key discovery. It will address an important roadblock that is currently preventing the development of genome editing-based therapies.
This published PCT application further contains in vivo data demonstrating the efficacy of Defence’s Accum™-based technology. Accum™ has shown to be efficient in transforming traditionally non-antigen presenting and immune quiescing cells, like mesenchymal stem cells (MSCs), into efficient antigen presenters and immunostimulatory cells in the form of an anti-cancer cellular vaccine.
The discovery led to the development of Defence’s potent second-generation MSC-based therapeutic ARM vaccine for lymphoma, which yielded extraordinary results in a pre-clinical study. The development is progressing into Phase I clinical trials in the near future.
“The filing and publication of this new PCT application is another major steppingstone towards the commercialisation and approval of innovative therapeutic and prophylactic candidates currently in Defence’s arsenal. The newly published PCT application adds to Defence’s rapidly-growing patent portfolio, as the Defence team continues to march toward their goal of bringing their innovative therapeutic technologies to market and making a lasting global impact on the lives of patients,” said Sébastien Plouffe, CEO and President of Defence.
