Hieff Trans PEI Transfection Reagents—R&D and GMP Grade for Viral Vector Production

Cell therapy refers to the use of biotechnology methods to obtain cells with specific functions, which are then enhanced through processes such as ex vivo expansion and specialized cultivation. These cells are endowed with augmented immune responses, pathogen and tumor cell killing capabilities, and other functions to achieve the therapeutic goals for certain diseases. Gene therapy refers to the therapeutic approach of modifying individual gene expression or repairing defective genes through methods such as gene addition, gene modification, and gene silencing. The ultimate aim is to cure diseases by rectifying abnormal genes.

Viral Vector Production and Expression Solution

The core of Cell and Gene Therapy (CGT) drug production lies in the expression of viral vectors, a process that typically involves both upstream vector expression and downstream purification. Vector production involves highly complex processes, presents significant challenges, and often has long preparation cycles. Consequently, the global GMP production capacity for viral vectors is approaching a bottleneck, posing a major obstacle to the entire gene therapy industry's development. The development, scale-up, and GMP production of gene therapy vectors involve intricate production systems and stringent quality control systems. These encompass processes such as strain, cell, and virus banks establishment, large-scale E. coli fermentation, cell culture processes, virus harvest and purification processes, aseptic processing, and formulation and filling processes.

AAV Production Costs

Apart from fixed asset investments (such as equipment, cleanrooms, etc.) and labor costs, the main expenses arise from material consumption in the upstream and downstream processes. Upstream costs mainly involve raw materials like plasmids, culture media, transfection reagents, and nucleases; downstream costs mainly include chromatography resins, quality control expenses, and more.

Cationic Polymer Carrier

Cationic polymers (polymers) include polyethylenimine (PEI), poly(beta-amino ester) (PBAE), chitosan, polyacrylamide (PAH), diethylaminoethyl dextran (DEAE-dextran), poly(amidoamine) dendrimers (PAMAM), and others. The common principle of cationic polymer transfection involves complexing DNA under physiological pH conditions to prevent degradation by DNase. Subsequently, these complexes attach to the cell membrane and are taken up by endocytosis, followed by rupture and release of DNA into the cytoplasm to exert their intended functions. The primary distinction between cationic polymers and cationic lipids lies in the absence of hydrophobic portions in cationic polymers, making them completely water-soluble and allowing for convenient chemical modifications.

Hieff Trans® PEI Transfection Reagents

 Transfection of Single Plasmid

Virus Titer Data 

1. LV Production

2. AAV2 Production

3. AAV5 Production

 4. RV Production

 Product Information