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mRNA for Protein Replacement Therapy

Background mRNA-based Protein Replacement Therapy Services Highlights FAQs

Background

Creative Biolabs is a global company that focuses on messenger ribonucleic acid (mRNA) studies. With our extensive experience and advanced platform, we can provide a series of mRNA-based protein replacement therapy services based on state-of-the-art technologies. We are proud to partner with our clients on the journey of bringing novel mRNA-based protein therapies to the clinical application.

  • Introduction to Protein Replacement Therapy

Proteins have been considered as key molecules in humans, and they have proven their significant roles in regulating physiological and biological reactions, protecting the human body from viral and bacterial infections. In the past few years of studies, a wide variety of human diseases have been associated with different proteins. Moreover, most natural and modified proteins have been generated by various advanced technologies, such as genetic engineering and protein engineering, to be further expressed by different systems, including phage display technology.

Also, therapeutic proteins have been broadly used for developing novel therapies against human diseases. Among them, protein replacement therapy has been used as an effective method for replacing the dysfunctional proteins or missing proteins in patients. For instance, pilot studies have demonstrated that replacing the defective collagen VII (C7) protein is essential to improve the performance of wound healing in patients with recessive dystrophic epidermolysis bullosa (RDEB). As a result, protein replacement therapy has become a potential therapy for a wide array of rare monogenic diseases, blood disorders, and metabolic disorders.

  • mRNA-based Protein Replacement Therapy

Currently, mRNA-based therapeutics has shown its great potential in the treatment of various human diseases, especially for malignant tumors. mRNA delivery systems have been regarded as safe and transient methods with a high transduction capacity in disease therapies. In recent studies, a new mRNA-based protein replacement therapy has been designed based on replacing the uridine residues in mRNA with nucleoside pseudouridine. The results have indicated that this therapy can interrupt innate immune responses caused by RNase and Toll-like receptors, reducing the expression level of various cytokines and avoiding the immunogenicity during treatment.

mRNA Services for Protein Replacement Therapy

As a professional expert in the field of mRNA therapeutics development, Creative Biolabs offers a one-stop mRNA-based platform for our clients, to design and synthesize suitable mRNA constructs, to generate safe and effective mRNA-based protein replacement therapies, as well as to evaluate the properties of mRNA-based therapies by using a panel of immunoassays. Besides, the mode of delivery routes can be assessed by our labs to avoid protein metabolism or clearance before entering the target cells and tissues. For example, in vitro transcribed (IVT) mRNA has worked as a protein replacement therapy for the heart. The data has suggested that this mRNA therapy carrying VEGF-A into mice heart can strongly increase the survival rates (80% survival) of mice in different heart disease models. Up to now, several protein replacement therapies have been generated by our labs, and several of them have been used in many large scales of rare monogenic disease models.

Diagram providing an overview of IVT mRNA-based therapeutics.Fig.1 An overview of IVT mRNA-based therapeutics.1

By continuing to grow in response to the requirements of our clients, Creative Biolabs is dedicated to exploring novel mRNA therapeutics to offer the most comprehensive, integrated portfolio of mRNA products and solutions. The members in our labs have originated from a rich academic research background, bringing their experience into practice. If you are interested in our services, please feel free to contact us for more information.

Inquire About Our Services

Highlights

  • Advanced Therapeutic Protein Development: We develop advanced mRNA-based protein replacement therapies for diseases, leveraging cutting-edge genetic and protein engineering technologies.
  • Custom mRNA Constructs: Creative Biolabs designs custom mRNA constructs to replace dysfunctional proteins, providing personalized therapeutic solutions for specific diseases.
  • Innovative mRNA Delivery Systems: We utilize advanced delivery systems like IVT mRNA for targeted, efficient delivery of therapeutic proteins, enhancing treatment effectiveness.
  • Expert mRNA Therapy Evaluation: Our labs conduct comprehensive immunoassays to ensure the safety, efficacy, and quality of mRNA-based protein replacement therapies.
  • Commitment to Innovation: Creative Biolabs continuously innovates in mRNA therapeutics, offering tailored solutions and a comprehensive portfolio to meet client needs.

FAQs

Q: What is mRNA-based protein replacement therapy?

A: RNA-based protein replacement therapy involves using messenger RNA to instruct cells to produce specific proteins that replace dysfunctional or missing proteins in the body. This method offers a transient and safe approach to treating diseases caused by protein deficiencies, such as rare monogenic disorders, by providing a temporary source of the needed protein.

Q: How does mRNA-based therapy differ from traditional protein replacement therapies?

A: Traditional protein replacement therapies often involve directly administering the protein to patients, which can be costly and less efficient. In contrast, mRNA-based therapy delivers mRNA that encodes the protein, allowing the patient's cells to produce the protein themselves, leading to potentially higher efficacy and reduced immunogenicity.

Q: What are the advantages of mRNA-based protein replacement therapy?

A: This therapy offers several advantages, including a lower risk of immune response, efficient and localized protein production, and the ability to quickly adapt to different proteins as needed. It also provides a more stable and controlled delivery method, with the mRNA being designed to optimize protein expression while minimizing potential side effects.

Q: Which diseases can be targeted by mRNA-based protein replacement therapy?

A: mRNA-based protein replacement therapy is particularly effective for treating rare monogenic diseases, metabolic disorders, and blood disorders where specific protein deficiencies are the underlying cause. It can also be applied in regenerative medicine and to improve outcomes in conditions like heart disease by promoting tissue repair through protein expression.

Q: How does Creative Biolabs support the development of mRNA-based protein replacement therapies?

A: Creative Biolabs offers comprehensive services, including mRNA design, synthesis, delivery system development, and preclinical evaluation. They provide tailored solutions for each project, ensuring that the mRNA constructs are optimized for stability, expression, and safety, with rigorous testing to meet the therapeutic needs of each client.

Q: What challenges exist in developing mRNA-based protein replacement therapies?

A: Challenges include ensuring the stability of mRNA, avoiding degradation by RNases, controlling the immune response, and optimizing delivery to target cells. Creative Biolabs addresses these challenges through advanced mRNA modification techniques, careful design of delivery systems, and thorough preclinical testing.

Q: How is the safety of mRNA-based protein replacement therapies ensured?

A: Safety is ensured through the use of non-integrating mRNA, reducing the risk of insertional mutagenesis. Additionally, mRNA constructs are designed to minimize immune activation, and comprehensive preclinical studies are conducted to evaluate potential side effects and ensure the therapy is both effective and safe.

Featured mRNA Products

Hot IVT Vectors

Cat. No Product Name Promoter
GTVCR-WQ19MR IVTScrip™ pT7-VEE-mRNA-Anti-H1-0, (131)I-chTNT-1/B Vector T7
GTVCR-WQ22MR IVTScrip™ pSP6-VEE-mRNA-Anti-H1-0, (131)I-chTNT-1/B Vector SP6
GTVCR-WQ24MR IVTScrip™ pT7-VEE-mRNA-Anti-KDR, 1121B Vector T7
GTVCR-WQ25MR IVTScrip™ pSP6-VEE-mRNA-Anti-KDR, 1121B Vector SP6
GTVCR-WQ28MR IVTScrip™ pT7-VEE-mRNA-Anti-EGFR, 11F8 Vector T7

Hot IVTScrip™ mRNA Transcript

Cat. No Product Name Type
GTTS-WQ15MR IVTScrip™ mRNA-Anti-S, 2130(Cap 0, 5-Methyl-CTP & Pseudo-UTP, 120 nt-poly(A)) Antibody
GTTS-WQ16MR IVTScrip™ mRNA-Anti-S, 2130(Cap 1, 5-Methyl-CTP & Pseudo-UTP, 120 nt-poly(A)) Antibody
GTTS-WQ17MR IVTScrip™ mRNA-Anti-S, 2130(Cap 0, 5-Methyl-CTP & Pseudo-UTP, 30 nt-poly(A)) Antibody
GTTS-WQ18MR IVTScrip™ mRNA-Anti-S, 2130(Cap 1, 5-Methyl-CTP & Pseudo-UTP, 30 nt-poly(A)) Antibody
GTTS-WQ19MR IVTScrip™ mRNA-Anti-S, 2130(Cap 0, Pseudo-UTP, 120 nt-poly(A)) Antibody

Featured mRNA Services

Reference

  1. Van, H. L.; et al. How mRNA therapeutics are entering the monoclonal antibody field. Journal of Translational Medicine. 2019, 17(1). Distributed under Open Access license CC BY 4.0, without modification.
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