Messenger ribonucleic acid (mRNA) is a molecule that contains the instructions to direct cells to make proteins that support different physiological functions, which play crucial roles in growth, development, inheritance and variation. Theoretically, mRNA can express any protein, therefore can be harnessed to treat almost any protein-related diseases. mRNA can potentially provide transformative therapeutics to conquer diseases that are incurable by traditional modalities.

Revolutionary therapies bring life hope to patients

When mRNA is delivered in cells to produce proteins, it becomes a therapy to treat diseases.

Developing mRNA-based therapeutics is a marathon

The scientific research and drug development efforts on mRNA have persisted for over half a century since the mRNA molecule was discovered in the 1960s. The stability, delivery efficiency, and readiness for mass manufacturing of mRNA molecules are three main obstacles to the development of mRNA-based therapeutics. The long history of innovative mRNA drug discovery is both a marathon and a relay race. Looking back farther in history of mRNA drug research and development, each hard-earned breakthrough has paved the way for this medical revolution. History makes us better history makers.

Delivering transformative therapies with our advanced mRNA technologies

We have established a leading mRNA technology platform including mRNA design, synthesis and modification to ensure the stability, safety, and effectiveness of mRNA molecules, as well as proprietary ionizable cationic lipids and advanced dynamic precision mixing technologies for the large-scale manufacturing of mRNA-based drug products.

Our technology platform covers the entire drug development life cycle from concept to market, including target selection, sequence design, plasmid preparation, in vitro mRNA synthesis, lipid molecule design, formulation process development, clinical research and GMP production.

The innovation of our platform technology is an engine that drives the development of mRNA medicines. Based on this platform, we have developed a series of mRNA vaccines for COVID-19, including the first China-made mRNA vaccine approved to the market. With increasing capabilities, we continue to expand our product pipeline in fields of infectious diseases control, immune-oncology, protein replacement therapy and many other diseases. We aim to bring more revolutionary therapies to patients suffering untreatable and emerging diseases.

mRNA design, synthesis, and modification
Synthetic mRNA molecules are the active drug substances of mRNA therapeutics. They can guide body cells to generate antigens to stimulate immune response against pathogens, immune modulators to fight against cancer, and functional proteins for the treatment of rare diseases. The structure of mRNA can be divided into five components, including the 5'cap, the 5' and 3' untranscriptional region (UTR), the open reading frame for the gene of interest, and the poly(A) tail. Each component is crucial to the stability and quality of the final product.
Our team has a wealth of experience in mRNA sequence design, in vitro transcriptional synthesis, modification, and purification, which enables us to go through the whole process from raw protein sequences to high-quality mRNA drug substances. We employ AI-based computational biology, high-throughput screening, and other means to keep improving our ability in mRNA sequence design and optimization, helping to turn novel concepts into real products.
Our mRNA design and synthesis capabilities have been further proven in the development of a series of COVID-19 mRNA vaccines, as we have designed the sequence and prepared the mRNA vaccines in just a few weeks for preclinical studies. This highlights the efficiency and capacity of Abogen's mRNA platform.
Lipid nano particle (LNP) delivery technology
mRNA is a negatively charged macromolecule which is difficult to pass through the negatively charged lipid bilayer of the cell membrane. In addition, mRNA molecules can be easily degraded by nucleases in the environment and body fluids. Endosomal release is another obstacle to mRNA delivery. An efficient and safe delivery system is critical for the development of mRNA medicines.
The delivery system is one of the hurdles in the development of mRNA vaccines. We are among the quite few mRNA companies with proprietary LNP delivery technology in the world. As the most advanced carrier for mRNA delivery in clinical practice, LNP is composed of ionizable lipids, neutral lipids, polyethylene glycol lipids, and cholesterol in different proportions. It has many advantages, such as high encapsulation efficiency, effective transfection, and strong tissue penetration, allowing for efficient delivery of therapeutic agents, low cytotoxicity and immunogenicity. On a global level, both of the two commercially available mRNA vaccines today are built with the LNP delivery technology.
Abogen's LNP system contains proprietary ionizable lipids, which have superior delivery efficiency and can ensure low toxicity and high expression of mRNA in human body. Our mRNA vaccines for COVID-19 are also powered by our proprietary LNP delivery technology.
Dynamic and precise mixing technology
mRNA-LNP is formulated by mixing the lipid ethanol solution and mRNA solution according to the appropriate flow rate and mixing ratio. Encapsulation rate and polydispersity index (PDI) are the primary indicators for evaluating quality, which requires precise control over such parameters as LNP components, particle size, flow rate, and fluid morphology at a high speed without compromising the quality. Very few companies possess these key technologies. Our in-house developed dynamic and precise mixing technology produces uniformly distributed LNPs with over 90% encapsulation efficiency, in just one step. The high-speed mixing reduces the solubility of lipids to help form uniform nanoparticles, which also ensures consistent quality and saves on production costs. The production process is scalable, and the mixing process and ultrafiltration process are fully compliant with the GMP requirements.