Applications - RIBOPRO

Examples of what
can be done with mRNA

Corona vaccines

At the moment, the most well-known example of mRNA in the clinical setting is the use as vaccine against SARS-COV-2 (Corona virus). Before these vaccines, no mRNA medicine or vaccine had reached the market, mainly due to the mRNA technology being relatively new. Only recently, a combination of de-immunization of mRNA and improved mRNA-delivery technology, allowed effective use of mRNA.

These Corona vaccines have shown the robust immunological responses when expressing non-self proteins in a manner that mimics a viral infection (non-self proteins produced by autologous proteins). In addition, they proved that mRNA is a relatively easy technology to develop new vaccines with, because mRNA production is robust, regardless of sequence, whereas virus or protein production often require elaborate optimization and characterization.

mRNA-based antibody therapeutics

Antibodies are currently a popular strategy for the development of novel therapeutics. Over a hundred therapies have already been approved for the treatment of a wide range of conditions, including auto-immune diseases, cancer, infectious diseases, allergies, and neurological disorders. As the traditional manufacturing of antibodies is costly and time-consuming, there new strategy emerging that uses mRNA for endogenous production of the desired antibodies.

mRNA can circumvent several of the challenges of protein-based antibody production. It contains consistent properties which removes the need for a tailored purification process for each therapeutic antibody. Moreover, endogenous production prevents the introduction of aberrant post-translational modifications and allows the antibody to reach intracellular targets. Lastly, mRNA-based expression enables the antibody to be detectable and active for multiple days or even weeks, in contrast to a single peak of active protein after antibody injection.

Currently, mRNA-based antibody therapeutics are already being explored in multiple (pre)-clinical studies. Naturally, it brings its own set of challenges – such as finding a suitable delivery method or the synthesis of long mRNA sequences – but recent advances in the field make mRNA-based antibody therapeutics a cost-effective, efficient, and flexible alternative to the traditional protein-based methods.

mRNA therapies for rare metabolic disorders

Rare metabolic diseases are caused by mutations in genes that encode enzymes involved in metabolism, resulting in the accumulation of toxic substances in the body. Traditional approaches for their treatment, such as enzyme replacement therapy, have limitations that can make them ineffective for some patients. mRNA technology offers a new approach to treating these conditions, with several advantages over the traditional approaches.

mRNA technology allows for the direct delivery of the missing enzyme to affected cells, potentially improving the effectiveness of the treatment. Additionally, mRNA can be customized to encode any protein, including enzymes that are missing in rare metabolic diseases. It has the potential to be used for a wide range of metabolic disorders, and can be adapted as new treatments are developed. Because mRNA technology uses mRNA to encode the missing enzyme, there is a lower risk of an immune reaction than with enzyme replacement therapy as well.

Several rare metabolic diseases, such as phenylketonuria, methylmalonic acidaemia, and ornithine transcarbamylase (OTC) deficiency, are being explored as potential candidates for mRNA-based treatments. mRNA will then be delivered to patients to encode the missing enzyme, with promising results in preclinical studies.

While these mRNA-based treatments are still in development, it offers a promising approach to treating rare metabolic diseases as it has the potential to bring about a customizable, targeted, and potentially curative treatment option.

Next-generation vaccines