Enzyme replacement therapy development and production services
Enzyme replacement therapy (ERT) is a revolutionary treatment approach primarily used to treat inherited metabolic diseases caused by the loss or deficiency of specific enzymes. These diseases are often caused by genetic mutations that prevent the synthesis of key enzymes, leading to the accumulation of toxic substrates in cells and progressive organ damage. The core principle of ERT is to replace the missing or ineffective natural enzymes in the patient's body through intravenous infusion of functional recombinant enzyme preparations. These exogenous enzymes can be delivered to damaged cells and organelles, breaking down the accumulated substrates, alleviating symptoms, halting disease progression, and improving patients' quality of life.
With the rapid advancement of biotechnology, ERT has become an important treatment option for numerous rare diseases, including Gaucher disease, Fabry disease, and Pompe disease. However, developing highly effective, stable, and immunogenic therapeutic enzymes remains a significant scientific and engineering challenge—an area we specialize in. Creative Enzymes Diagnostic committed to translating cutting-edge enzyme science into life-saving therapies. Leveraging cutting-edge enzyme engineering technologies and GMP-compliant production systems, we provide comprehensive enzyme replacement therapy solutions, from target discovery to commercial supply, to our global partners, helping to combat inherited metabolic diseases.
Creative Enzymes Diagnostic provides one-stop services covering the entire development cycle of enzyme replacement therapy, ensuring that every step of the project is accurate and efficient.
| Target discovery and validation | Disease mechanism analysis | Based on the target disease, conduct in-depth analysis of its pathological mechanisms and key enzyme targets |
|---|---|---|
| Enzyme characterization studies | Fully characterize the target enzyme's natural properties (optimal pH, temperature, kinetic parameters, etc.) | |
| In vitro functional validation | Establish cell models to verify the activity and efficiency of the recombinant enzyme in in vitro substrate degradation | |
| Enzyme engineering and optimization | Recombinant expression system development | Utilize eukaryotic expression systems such as mammalian cells (e.g., CHO and HEK293) to ensure proper enzyme folding and glycosylation |
| Protein engineering | Apply advanced technologies such as site-directed mutagenesis and directed evolution to rationally design enzymes | |
| Process development and scale-up | Cell line development | Develop high-yielding, stable expression therapeutic cell lines |
| Upstream processing | Optimize culture conditions from shake flasks to large-scale bioreactors | |
| Downstream purification | Establish efficient and robust purification processes to ensure product purity, yield, and activity | |
| Formulation development | Develop enzyme formulations suitable for long-term storage and clinical use, and thoroughly investigate their physicochemical stability and biological activity | |
| Analytical method development and quality control | Activity analysis | Develop sensitive and specific methods to detect the enzyme's biological activity |
| Physicochemical analysis | Comprehensively characterize key quality attributes such as enzyme purity, molecular weight, isoelectric point, and glycosylation profile | |
| Stability studies | Perform accelerated and long-term stability testing to support product expiration date determination | |
| GMP-compliant manufacturing | We have cleanrooms and production facilities that meet cGMP standards, and can provide high-quality enzyme APIs or finished formulations for preclinical research, clinical trials, and commercial supply | |
Figure 1. General Service process.(Creative Enzymes)
Whether you're in the early stages of R&D or need a reliable GMP manufacturing partner, we're ready to support you. Contact our business development team today to discuss your specific project needs!
