mRNA protein replacement therapy is a novel drug development technology that utilizes artificially synthesized mRNA to instruct human cells to produce required proteins in vivo. Compared to traditional protein drugs, it offers longer expression duration, lower immunogenicity, and better sustained efficacy, making it suitable for pharmacological research on proteins that are challenging to synthesize ex vivo. It has broad application prospects in various fields including genetic metabolic diseases (such as hyperuricemia), cardiovascular diseases (such as he EnoBio provides a fully integrated, proprietary intellectual property solution from drug target design to clinical sample production and regulatory submission. Clients can choose from a range of integrated or modular/technical solutions according to their specific needs, including AI-assisted protein design platform (DMC.AI platform), LNP delivery systems, non-LNP delivery systems, as well as cap analogs, UTR, and poly A tails for enhanced expression optimization. Leveraging proprietary intellectual property technologies, critical raw materials, and a research and development production equipment supply chain, EnoBio not only addresses the comprehensive cost challenges of mRNA protein replacement drugs but also meets diverse demands for mRNA protein replacement drug development and related pharmaceuticals with full intellectual property control.art failure), and degenerative diseases (such as osteoarthritis).
Integrated solution for mRNA therapeutics as protein substitutes
case study:
| Application | Characteristic Analysis | Key Capability Elements for Characteristics |
|---|---|---|
| mRNA Therapeutic for Osteoarthriti | Local Administration, Chondrocyte Targeting, Intellectual Property Needs | Cap Structure Optimization (Tissue Specificity) |
| Non-LNP Delivery Technology | ||
| High-Expression and High-Stability mRNA Design | ||
| Fully Proprietary Intellectual Property | ||
| mRNA Therapeutic for Hyperuricemia | Liver Targeting, Target Protein Nonexistent in Human Body, Intellectual Property Requirements | Protein Humanization Design |
| Cap Structure Optimization (Tissue Specificity) | ||
| Highly Safe Liver-Targeted LNP Delivery System | ||
| Comprehensive Intellectual Property Ownership | ||
| mRNA Therapeutic for Hyperglycemia | Liver Targeting, Long-acting Requirement, Intellectual Property Needs | Protein Affinity and Long-acting Design |
| Cap Structure Optimization (Tissue Specificity) | ||
| Highly Safe Liver-Targeted LNP Delivery System | ||
| Comprehensive Intellectual Property Ownership |
Development process:
| Target protein screening and drug design |
Data mining Protein affinity and long-acting design Protein humanization design Homologous protein sequence design High-expression and high-stability mRNA design mRNA sequence CDS optimization Enhanced expression UTR elements* |
| mRNA optimization and synthesis |
Tissue-specific mRNA cap analogs* Enhanced expression and stability with poly A tails* Design and synthesis of multi-epitope mRNA sequences (encoding multiple antigens) Fully proprietary mRNA synthesis without IP risk |
| LNP delivery and formulation development |
Tissue- or organ-targeted LNP delivery system* LNP formulation DOE screening for target mRNA and indications mRNA-LNP lyophilization technology* Research-grade mRNA-LNP encapsulation equipment* |
| Preclinical safety evaluation and pharmacokinetic assessment |
Protein expression detection Animal model construction Drug efficacy evaluation Preliminary safety assessment |
| Process development and GMP-level production |
High-genetic stability plasmid engineering bacteria* Plasmid Tier III library construction, process development, and GMP production mRNA bulk process development and GMP production Lyophilized formulation process development and GMP production* Quality method development and research GMP-like raw material production and supply* Industrial-scale mRNA-LNP encapsulation equipment* |
| Safety evaluation and regulatory submission |
Toxicology evaluation In vivo distribution characterization Quality method development and research Pharmacology data writing IND submission service |
* Available proprietary intellectual property solutions