mRNA for Protein Replacement Therapy

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mRNA for Protein Replacement Therapy

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.

Disease classes targeted by protein replacement therapies. Fig.1 Disease classes targeted by protein replacement therapies. (Crunkhorn, 2013)

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.

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.

An overview of IVT mRNA-based therapeutics. Fig.2 An overview of IVT mRNA-based therapeutics. (Van, 2019)

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.

Reference

  1. Crunkhorn, S.; et al. Regulatory watch: enhanced chance of success for protein replacement therapies. Nat Rev Drug Discov. 2013, 12(6): 414.
  2. Van, H. L.; et al. How mRNA therapeutics are entering the monoclonal antibody field. Journal of Translational Medicine. 2019, 17(1).
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