Solution of Gene Editing mRNA Technology

CRISPR-based gene editing technology enables efficient and convenient gene knockout, gene insertion, base editing, and is widely used in protein labeling, gene therapy, molecular breeding, disease model construction, and other fields. In early animal gene editing, AAV and lentivirus were used to deliver CRISPR systems, which were costly and raised safety concerns. Long-term expression of nucleases such as Cas9 may also lead to more off-target edits. With the maturity of mRNA technology, LNP-encapsulated mRNA and sgRNA targeting Cas9 can achieve targeted and efficient gene editing. Due to the transient expression of mRNA, the possibility of off-target editing can be reduced. For crop breeding, mRNA technology can achieve non-transgenic gene editing.

We offer a complete solution from gene editing scheme design to final outcome evaluation, enabling customers to easily achieve various gene editing tasks including knockout, knock-in, and base editing. This solution caters to the diverse mRNA gene editing tool requirements across different research fields, empowering customers to generate high-level scientific research outcomes efficiently.

case study:

Application Characteristic Analysis Key Capability Elements for Characteristics

1. Gene Therapy

2. Disease Models

3. Antiviral Therapy

4. Protein/RNA Labeling

5. Molecular Breeding

Design and Synthesis of Cas9 mRNA, sgRNA, etc Optimization of Cas9 mRNA and other components according to customer requirements, followed by in vitro transcription (IVT) synthesis of designed mRNA and sgRNA, aimed at achieving optimal gene editing efficiency.
mRNA Delivery Tissue-specific and organ-targeted LNP delivery system for efficient targeted delivery, in addition to other delivery methods such as gene gun, protoplast transfection, and microinjection.
Associated Detection and Evaluation We provide comprehensive analytical services including sequencing, mRNA integrity assessment, capping efficiency determination, mRNA sequence consistency verification, lipid identification and quantification analysis, mRNA content and purity determination, encapsulation efficiency measurement, as well as in vitro and in vivo activity assays, small animal fluorescence imaging, animal modeling,animal experiments, gene editing efficiency evaluation, gene editing, and genotype and phenotype confirmation for both in vitro and in vivo assessments.

1. Lyophilized mRNA-lipid nanoparticle vaccines with long-term stability and high antigenicity against SARS-CoV-2. . 2023, 9(1): 9

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

LNP formulation DOE screening for target mRNA and indications

mRNA-LNP lyophilization technology*

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

Plasmid Tier III library construction, 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

Pharmacology data writing

IND submission service characterization

Quality method development and research

* Available proprietary intellectual property solutions