- One-stop service for mRNA design/synthesis, LNP formulation, and protein expression evaluation
- Aquisition of the lead mRNA/LNPs with confirmed in vivo expression within three months the fastest
- Smooth workflow to initiate further non-clinical evaluation after determination of the optimized mRNA/LNP
Efficient design, synthesis, and evaluation of mRNA
▶Quick optimization of mRNA sequences
– Original design methods of CDS, UTR, and polyA tail
– Minimization of rare codons
▶Successful mRNA synthesis and QC technology
– Established and accurate Drug Substance manufacturing technologies licensed from Arcturus Therapeutics
– Aditional evaluation of optimized mRNA/LNP upon the customerʼs request
▶Cell-based evaluation of protein expression levels
– Index the translational efficiency in culture cells due to sequence differences
Robust LNP formulation and evaluation technology
▶Successful LNP formulation
– Established LNP formulation by micro-flow
– Characterization capability of LNP
▶Diverse in vitro and in vivo evaluation capability
– In vitro evaluation and subcellular localization
– In vivo evaluation and tissue distribution by IVIS imaging system
– Immune response caused by mRNA/LNP
– Efficacy using various disease models
▶Concrete support by experts
– Evaluation by each technology experts
– One-stop task control by your product manager
mRNA design, synthesis, and evaluation service
ARCALIS designs, synthesizes, and evaluates about ten mRNAs as an initial candidate group and determines candidate sequences to proceed to LNP formulation.
▶mRNA sequence design
Designs mRNA sequences, which are expected to express target proteins efficiently according to our original algorism and experimental data, and selects the most appropriate manufacturing methods, based on their mRNA sequences.
▶mRNA synthesis and quality control (QC)
Template DNA synthesis based on our original design, followed by in vitro transcription (IVT) and purification to produce a group of mRNAs. As a basic QC, mRNA yield (UV) and mRNA integrity (CE) will be provided.
▶Selection of candidate mRNAs by in vitro evaluation
Will evaluate protein expression level by candidate mRNAs in culture cells. Alternatively, ELISA and/or western blotting will be conducted depending on customers’ presence or absence of evaluation methods.
LNP formulation, in vitro or in vivo evaluation service
Axcelead DDP supports the evaluation of mRNA delivery by LNP.
▶LNP formulation
Will encapsulate mRNA synthesized by ARCALIS with lipids selected by customers
▶In vitro evaluation
Will evaluate mRNA transfection efficiency and target protein expression in target cells and immune response analysis based on cytokine production using human peripheral blood mononuclear cells (PBMC).
▶In vivo evaluation
Will evaluate the efficacy, body distribution of mRNA by IVIS imaging system, target protein expression, distribution in tissue, immune response, and safety.
