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mRNA Therapeutics as Protein Replacement

mRNA Therapeutics as Protein Replacement 日本

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mRNA Therapeutics as Protein Replacement

The human body can be the utilized by mRNA to translate and encode therapeutic proteins like transmembrane, intracellular, intramitochondrial, and secreted proteins. mRNA-based protein replacement therapy provides a viable alternative cure for various diseases.

Clinical trials have demonstrated that mRNA-based therapeutics can be used in myocardial ischemia (AZD8601, VEGF mRNA), ornithine transcarbamylase deficiency (ARCT-810, OTCase mRNA), methylmalonic acidemia (mRNA-3705, MUT mRNA), propionic acidemia (mRNA-3927, PCCA/B mRNA), cystic fibrosis (MRT-5005 and VX-522, CFTR mRNA).

In addition, mRNAs directly encode antibodies (e.g., neutralizing antibodies, antibody fragments, and nano-antibodies) for the prevention and treatment of viral infections and cancer. These antibodies specifically recognize viruses, bacteria, and cancer /tumor cells and bind them via long variable chains, which turns out to be useful for the prevention and treatment of SARS-CoV-2, HIV, as well as animal models and human tumors.

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Drug Substance, mRNA

0.1~10 mg (mRNA)

Preclinical research such as cell transfection, Analytical method development, Pre-stability studies, Formulation development

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GMP, Sterility

Drug Substance, mRNA

10 mg~70 g

Investigational new drug (IND), Clinical trial authorisation (CTA), Clinical trial supply, Biologic license application (BLA), Commercial supply

Drug Product, LNP-mRNA

5000 vials or pre-filled syringes/ cartridges

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