RNA-enabled protein replacement model
Protein replacement therapies aim to restore or supplement missing, deficient, or dysfunctional proteins in patients with rare diseases, metabolic disorders, enzyme deficiencies, and other protein-loss or protein-insufficiency conditions. Beyond traditional recombinant protein administration, nucleic acid-based approaches such as mRNA, circRNA, saRNA, and DNA/RNA-enabled strategies can instruct patient cells to produce therapeutic proteins in vivo, creating new opportunities for transient, repeatable, and potentially more flexible protein replacement.
Key development questions
For RNA- or LNP-enabled protein replacement programs, the key questions are whether the payload can achieve sufficient protein expression, whether expression duration matches the therapeutic need, whether the delivery system can reach the intended tissue or cell type, and whether repeat dosing can be supported with an acceptable safety and immunogenicity profile.
RNA payload and expression strategy
CATUG provides a comprehensive RNA payload platform covering mRNA, circRNA, and saRNA, allowing clients to evaluate different expression profiles and durability strategies for protein replacement applications. For programs where expression duration is critical, circRNA or saRNA may offer alternative development routes beyond conventional mRNA, depending on the target protein, required exposure window, tissue distribution, dosing strategy, and safety considerations.
Process safety and impurity control
Process safety and impurity control are central to RNA-enabled protein replacement CMC. CATUG has established RNA process development capabilities for dsRNA reduction, residual impurity control, RNA integrity optimization, and purification strategy development, helping clients improve payload quality, reduce process-related risks, and build more controlled manufacturing workflows for IND-enabling and clinical-stage programs.
LNP and tLNP drug product support
On the drug product side, CATUG supports LNP/tLNP formulation development and scale-up, including experience with MaxMix™ LNP processes designed to improve manufacturing scalability, process robustness, and transition from early formulation screening to GMP-oriented production. Our delivery-system experience includes multiple lipid chemistries, innovative lipid designs, and internal lipid IP development to support next-generation formulation strategies.
CATUG integrated support
CATUG supports protein replacement programs through plasmid template preparation, RNA drug substance manufacturing, LNP/tLNP drug product formulation, analytical development, GMP manufacturing, fill-finish, stability studies, and CMC documentation. Clients can engage CATUG for selected modules such as RNA DS, LNP DP, analytics, or GMP manufacturing, or for an integrated CMC workflow from early feasibility to IND-enabling and clinical-stage development.
Why CATUG for protein replacement
CATUG helps innovators translate therapeutic protein expression concepts into developable, scalable, and quality-controlled programs with clear paths toward clinical translation.
CATUG supports ex vivo cell therapy, stem cell, and iPSC-related programs by providing critical upstream CMC inputs, including plasmid DNA, RNA payloads, guide RNA, gene-editing materials, LNP materials, protein / nanobody reagents, analytical support, GMP-oriented manufacturing, and documentation. For ex vivo CAR-related programs, CATUG has project experience supporting both China and U.S. IND filings.
CATUG does not position itself as a full cell-processing CDMO. The focus is on high-quality, stage-appropriate materials and CMC inputs that enable cell engineering, reprogramming, transient expression, gene editing, LNP-enabled cell transfection, and downstream process development.
Supports CAR-T, CAR-NK, TCR-T, gene-edited immune cell therapy, and other engineered cell therapy workflows through flexible upstream nucleic acid, editing, LNP, and protein materials.
Supports reprogramming, differentiation, transient expression, gene editing, and iPSC-related process development through flexible, small-quantity, multi-material supply.
Designed for route-flexible, multi-material, low-volume, quality-controlled enabling material supply.
Plasmid-only, plasmid + RNA, or plasmid + RNA + LNP input strategies.
Flexible supply for feasibility, process development, and early-stage programs.
Project experience supporting China and U.S. IND filings for ex vivo CAR-related programs.
Avoids forcing early programs into oversized manufacturing packages too early.
CATUG connects plasmid, RNA, LNP, gene-editing, protein, nanobody, analytical, and documentation capabilities to support different cell engineering routes and enabling material needs behind ex vivo cell therapy, stem cell, and iPSC programs.
Supports different ex vivo cell engineering strategies, including plasmid-only supply, plasmid + RNA inputs for electroporation, and plasmid + RNA + LNP workflows for cell transfection or delivery feasibility studies.
Plasmid construction, supercoiled plasmid manufacturing, linearized IVT template preparation, mRNA / circRNA payload manufacturing, and RNA DS support for transient expression, gene editing, and cell engineering workflows.
Guide RNA, sgRNA-related workflows, donor-template-related support, gene-editing enzyme materials, and compatibility considerations for electroporation or LNP-enabled cell engineering workflows.
Selected functional proteins, enzymes, reprogramming-related materials, cytokines, growth factors, and protein reagents for early feasibility and process development.
Nanobody, antibody fragment, peptide, or other ligand-related material support for targeted delivery, immune-cell targeting, LNP-enabled cell engineering, and advanced therapy feasibility studies.
Analytical testing, identity and purity support, impurity control, CoA, batch records, release documentation, CMC source documents, and project experience supporting China and U.S. IND filings for ex vivo CAR-related programs.
CATUG supports cell therapy and stem-cell-related programs through flexible entry points, from research-grade feasibility materials to GMP-oriented inputs and CMC documentation for IIT, IND-enabling, and clinical-stage preparation.
Built for CAR-T, CAR-NK, TCR-T, and gene-edited immune cell programs where clients may need plasmid-only inputs, plasmid + RNA for electroporation, or plasmid + RNA + LNP materials for cell transfection, delivery feasibility, and IND filing-ready CMC input documentation. CATUG has supported ex vivo CAR-related projects for both China and U.S. IND filings.
Built for iPSC, stem cell, reprogramming, differentiation, transient expression, and cell-line engineering workflows requiring flexible small-quantity materials and stage-appropriate quality control.