The novel messenger ribonucleic acid (mRNA) therapeutics strategies have aroused great attention in many clinical settings, such as cancer treatment. To meet the challenging requirements, Creative Biolabs has built a team of experienced scientists with facilities and processes designed specifically to provide the best strategy and protocols customized to mRNA-based services. We have accomplished many projects and have delivered numerous novel or validated mRNA therapeutics in a range of therapeutic areas.
mRNA is usually created during the process of in vitro transcription (IVT) of various linearized pDNAs or PCR products. It is composed of a promoter domain (5' cap), 5' and 3' untranslated regions (UTRs), an open reading frame (ORF), as well as a poly(A) sequence. In general, the modification of UTR and ORF sequences can strongly improve the efficiency of mRNA synthesis. Meanwhile, the poly(A) sequence and 5' cap play an important role in mediating the process of mRNA translation. IVT mRNA is capable of regulating the activation of innate immunity. Furthermore, pilot studies have shown that IVT mRNA delivery can be achieved by both in vitro and in vivo transfection assays. The results have indicated that these methods can deliver enough target cells to trigger strong immune responses against various human diseases, such as malignant tumors.
Fig.1 Schematic representation of synthetic mRNA and its structural features. 1
Basing on the requirement of mRNA study in drug discovery, Creative Biolabs has generated a one-stop mRNA therapeutics development platform to assist in all areas of mRNA-based projects, ranging from the design and production of mRNA molecules, mRNA biology, mRNA formulation and delivery, mRNA immunogenicity evaluation, to protein engineering. Till now, a wide variety of therapeutic areas have been exclusively studied by using a panel of mRNA-based technologies. For instance, we have also established several strategies, such as reverse transcription PCR (qRT-PCR) and branched DNA, for analyzing engineered mRNA therapeutics. Furthermore, we provide a series of safety and efficacy assessment services for mRNA therapeutics, which is essential to reduce the cost and improve the success rate of drug development. Our services for mRNA therapeutics including but not limited to:
A: We provide services that include, but are not limited to, messenger RNA design, production, delivery optimization, and immunogenicity assessment.
A: It assesses the immune response triggered by mRNA therapeutics to ensure safety and efficacy.
A: We have experienced scientists and industry experts in technology and therapeutic development who can provide clients with a one-stop mRNA therapy development service, saving them time.
A: Services can be customized for specific therapeutic needs, including target selection, mRNA modifications, and delivery methods.
A: We utilize state-of-the-art facilities and experienced scientists to ensure high-quality services. And comprehensive documentation and support can be provided to meet regulatory standards for clinical development and approval processes.
This article discusses a novel two-nanoparticle mRNA delivery system designed for intracellular enzyme replacement therapy (i-ERT) to treat ornithine transcarbamylase deficiency (OTCD), a severe inherited metabolic disorder. This Hybrid mRNA Technology (HMT) system consists of an inert lipid nanoparticle and a polymer micelle, which together protect and deliver mRNA to the liver, targeting hepatocytes for efficient mRNA translation. The study demonstrates that HMT can normalize plasma ammonia and urinary orotic acid levels, providing a prolonged survival benefit in a murine model of OTCD. The HMT delivery method represents a promising non-viral approach for treating single-gene metabolic diseases with systemic administration.
Fig.2 The hybrid mRNA technology delivery system.2
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