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Therapeutics

mRNA-based therapeutics

An mRNA-based approach to therapeutics has recently emerged as an alternative to monoclonal antibody-based drugs, which often require complex production and purification processes, and aberrant post-translational modifications of the antibody.

With the mRNA-based approach to therapeutics, the genetic information of the antibody — not the antibody itself — is delivered. Transient gene transfer aims at administering the mAb-encoding nucleotide sequences in DNA or mRNA form, rather than the mAb protein itself, directly to patients. This allows for the in situ production of biologicals in a cost- and labor-effective manner, potentially for a prolonged period of time.

Although past research has been mainly focused on the development of plasmid DNA, the limitations associated with these “classical” approaches, and the recent improvements in stability and translatability of in vitro transcribed (IVT) mRNA have led to an increased interest in mRNA as a delivery vector. In addition to safer pharmaceutical properties, such as no risk of genome integration, the transient expression of mRNA-encoded antibodies enables a more controlled exposure, with more protein production during peak expression compared to naked plasmid DNA.

mRNA-based therapeutics

Enabling mRNA-based approaches to therapeutics

The BioXp™ system can be used to rapidly produce small-scale, biologically active mRNA for accelerated iteration of the design-build-test cycle for the identification of therapeutic candidates. In addition, a wide menu of on-demand, automated library synthesis enables the customer to further speed up iterative design-build-test cycles during the drug discovery and development continuum.

When library synthesis is used in combination with mRNA production, we estimate that a customer can reduce turnaround times by weeks or months when using the BioXp™ system for screening and optimizing the mRNA products that have the most desirable pharmaceutical properties.

BioXp™ system

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