Development of mRNA-based Therapeutics
Messenger RNA (mRNA) is a single-stranded ribonucleic acid transcribed from a strand of DNA that carries coding information for protein synthesis and is further transcribed and processed into a functional protein. Introducing mRNA as a vaccine or therapeutic agent makes it possible to produce almost any functional protein/peptide in the human body, which has great prospects for the prevention and treatment of many intractable or genetic diseases. Unlike DNA-based drugs, mRNA transcripts do not need to enter the nucleus to function and thus have relatively high transfection efficiency and low toxicity. Importantly, there is no potential risk of accidental infection or opportunistic insertional mutations in mRNA. Compared with transient traditional protein/peptide, mRNA is capable of continuous translation into proteins/peptides and long-lasting expression, and thus has broad potential for the treatment of diseases requiring protein expression with higher therapeutic efficacy.
The development of mRNA-based therapeutic drugs mainly includes mRNA design, synthesis, entrapment, pharmacodynamics, pharmacokinetics, safety evaluation, manufacture, and clinical trials. Design and synthesis of mRNA are key steps in mRNA-based therapeutics. mRNA contains five functional regions, including the 5' cap, the 3' Poly(A) tail, the open reading frame (ORF) flanking, and the 3' untranslated regions (UTRs), whose elements mediate mRNA translation efficiency and decay rate. Obtaining highly biologically active mRNA often depends on reliable design and preparation, in which high-quality mRNA-related enzymes and reagents are critical.
Drug Master File (DMF)
A Drug Master File (DMF) is a type of submission to the Food and Drug Administration (FDA) that can be used to provide confidential detailed information about the facilities, processes, or articles used in the manufacturing, processing, packaging, and storage of one or more drugs for human use. The information contained in the DMF can be used to support an investigational new drug application (IND), a new drug application (NDA), an abbreviated new drug application (ANDA), an export application, etc. DMF filing supports new drug applications by providing administrative departments with efficient access to drug manufacturing information, which saves costs and increases the efficiency of review while ensuring the traceability. The use of DMF filed materials can reduce the number of research required for drug application to shorten the drug registration period, allowing biopharmaceutical companies to devote more effort to the research and development of the drug itself.
DMF Filed Enzyme Products
Amerigo Scientific provides a range of mRNA-related raw enzymes and reagents to support the development and manufacture of mRNA therapeutics. The manufacturing and quality management procedures of our products are in accordance with GMP. FDA DMF filing for these products has been completed.
Products
| Product Name |
Description |
| T7 RNA Polymerase, GMP Grade (50U/μl)
|
The T7 RNA polymerase is produced in E. coli, which is used for in vitro mRNA synthesis. |
| Vaccinia Capping Enzyme, GMP Grade (10U/μl)
|
The vaccinia capping enzyme is capable to add the 7-methylguanosine capping structure (m7Gppp, Cap0) to the 5' end of the RNA. |
| mRNA Cap 2'-O-Methyltransferase, GMP Grade (50U/μl)
|
With S-adenosylmethionine (SAM) as a methyl donor, mRNA Cap 2´-O-methyltransferase can add a methyl group at the 2´-O of the first nucleotide at the 5' end of Cap0 to form Cap1. |
| Pyrophosphatase, Inorganic (yeast), GMP Grade (0.1U/μl)
|
Inorganic pyrophosphatase catalyzes the hydrolysis of inorganic pyrophosphate into two orthophosphates. |
| DNase I, GMP Grade (2U/μl)
|
Deoxyribonuclease I (DNase I) is an endonuclease that randomly degrades single- or double- stranded DNA, generating 5'-phosphodinucleotide and 5'-phosphooligonucleotide end-products. |
| BsaI, GMP Grade (10U/μl)
|
BsaI is a commonly used restriction endonuclease that recognizes the specific nucleotide sequence and cleaves DNA strands. |
| BenzoNuclease® Nuclease, GMP Grade (250U/μl)
|
BenzoNuclease® is a recombinant endonuclease that hydrolyzes internal phosphodiester bonds present between the nucleotides in DNA and RNA (single-stranded, double-stranded, linear, circular, and supercoiled). |
| RNase Inhibitor, GMP Grade (40U/μl)
|
RNase Inhibitor efficiently inhibits RNase A, B and C by forming a stable complex. |
Product Advantages
- GMP-grade, animal-free and ampicillin-free
- The use of our DMF filed products in the R&D phase can avoid the impact on manufacturing caused by the use of research-grade raw materials.
- All materials meet the requirements for the development and manufacturing of mRNA therapeutics.
- The only raw enzyme that has been validated in phase III clinical trials is available in 10 million doses per batch, for a total of 5 billion doses per year.
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