Arbutus Biologics Breaks Overseas Patent Barriers with Novel Cis-Based Circular RNA Platform

2024Year8Month21Day, Abogen atNucleic Acids ResearchPublished paper "Efficient circularization of protein-encoding RNAs via a novel cis-splicing system", reported its independently developed high-efficiencyRNACyclic Cis-Splicing System (hereinafter referred to as "CisSystem").The system prepares cyclicRNA (circRNA)It can significantly prolong protein expression, reduce innate immune activation, and offers flexibility in splice site design, demonstrating broad application potential.。

Figure1: Screenshot of published paper

In recent years, circularRNATechnology in the biopharmaceutical industry has attracted much attention, and several engineered circularRNAThe company was founded, and these companies have raised more than10Hundreds of millions of dollars in financing. Among them, the most notable one isFlagshipIncubatedSail Biomedicines (by Laronde, Senda Biosciencesmerged), has raised nearly 800 million US dollars in cumulative financing, while the Massachusetts Institute of Technology (MIT）Daniel AndersonFounded by ProfessorOrna Therapeutics In obtaining super3After raising hundreds of millions of dollars in financing, this year also announced the acquisitionReNAgade Therapeutics, further accelerating the development of its clinical products.

Abogen Biosciences, with its industry-leadingmRNAAnd the nano-delivery technology platform, accumulating rich R&D experience in the process of continuous development, successfully developed a completely new cyclicRNAThe technology platform demonstrates Abogen's strong R&D capabilities and innovative capacity in the field of nucleic acid drug development.

Abogen's New CircularRNAThe system has global independent intellectual property rights, breaking through overseas companies.PIESystem Patent Restrictions

Currently, more applications are used for in vitro synthesis.circ RNATechnology isPIELaw (Permuted Introns and Exons), representing companies such asOrna TherapeuticsAlready onPIESystemApply for patent protection, the core optimization work of this technology is carried out byMITCompleted by relevant experts and patented in multiple countries and regions.

This paper introducesCisSystemic CirculationRNA PCTThe patent has entered the public review stage, and the system has broken through overseas companiesPIERestrictions on system-level patents, particularly suitable for cyclicRNADevelopment and Industrial Application of Drugs. As reported in the paper, Abogen has developedCisThe system processes the precursorRNAOne end is embedded in the ribozyme core structure, eliminating the need for additional spacer elements between the ribozyme and the target gene, simplifying the cyclization process and improving efficiency.Thereby possessing the advantages of "seamless" and highly efficient translation, as well as overcoming the challenges in efficiently synthesizing high-quality...circRNAAnd other technical challenges。

Based onCisSystemic CirculationRNAWith Broad Prospects and a Promising Future

This study reportsCisThe system has high cyclization efficiency and isRNAThe bases and sequence composition at both ends of the molecule are basically unrestricted, enabling truly efficient and seamless cyclization. In addition,CisThe system design is simpler and more flexible, allowing for customized designs based on specific treatment needs and compatibility with different lengths.RNAMolecules, ultimately achieving moreLong-lasting protein expression, low innate immune stimulation，and potential tissue-specific expression, showing excellent application prospects in the fields of protein replacement for chronic disease treatment and immunotherapy, as well as in treatment strategies that may require repeated dosing.. Based on these advantages, the new typeCisThe system is expected to become an efficient preparation of high-quality cyclicRNA's breakthrough technology, better empowering circularRNADevelopment of nucleic acid drugs in the fields of infectious diseases, tumor vaccines, cell and gene therapy.

AbogenmRNADr. Shaojun Qi, Senior Researcher of Innovation (Nucleic Acid Science Department), stated:

"I deeply feel that the superior research conditions and innovative atmosphere provided by the company are key to our successful breakthrough in circular RNA technology."RNAAn important cornerstone of key technical bottlenecks. Here, every technological advancement and innovative achievement is the result of the company's continuous investment in R&D."

Dr. Peng Gao, Vice President of Abogen (and also the corresponding author of this paper), stated:

"After more than two years of hard work, our team finally broke through the cyclicRNAThe key technical bottleneck. Currently, related application research has achieved preliminary results, demonstrating the potential value and feasibility of this technology. We hope to quickly transform this original achievement into a practical solution to promote progress in medical research and industry." 

Original link:

Shaojun Qi, Huiming Wang, Guopeng Liu, Qianshan Qin, Peng Gao, Bo Ying, Efficient circularization of protein-encoding RNAs via a novel cis-splicing system, Nucleic Acids Research, 2024;, gkae711, https://doi.org/10.1093/nar/gkae711

About Us

Abogen Biosciences Co., Ltd. (referred to as "Abogen") is an innovative biopharmaceutical company dedicated to the research and development of messenger ribonucleic acid (mRNA) drugs. The company possesses industry-leading, proprietary mRNA and nanodelivery technology platforms and is one of the few mRNA drug developers in China with comprehensive value chain capabilities, covering all aspects from mRNA design, formulation development, to large-scale production. Abogen has established a robust product pipeline encompassing multiple fields, including infectious disease prevention and treatment (such as mRNA COVID-19 vaccines), cancer immunotherapy, and protein replacement therapies.