Drug intelligence / Profile preview

Nanoplasmid

Development stage
Unknown
Lead developer
Aldevron
Modality
Gene Silencing → Gene Therapies, Prophylactic Vaccines → Vaccines & Immunotherapeutics, DNA Vaccines → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration
Electroporation, Intramuscular Injection, Nanoparticle Tail Vein Injection, Intravesical Administration, Hydrodynamic Retrograde Intrabiliary Injection
01

Overview

Nanoplasmid is a next-generation plasmid vector system designed for cell and gene therapy applications. It features a small bacterial backbone of approximately 500 base pairs (compared to >2,000 bp in canonical plasmids) and utilizes an antibiotic-free RNA-OUT sucrose-based selection system. The platform is characterized by reduced transgene silencing, improved transfection efficiency, reduced post-transfection cellular toxicity, and enhanced transgene expression (up to 10-fold higher than traditional plasmids). Nanoplasmid employs an R6K replication origin instead of the traditional pUC origin, making it replication-incompatible with native organisms and reducing the risk of horizontal gene transfer. The vector has no coding capacity in its backbone, eliminating the risk of bacterial protein marker transfer to patients. It encompasses the performance benefits of minicircle vectors while maintaining high manufacturing yields and scalability. Applications include use as a transposon or CRISPR-Cas9 homology-directed repair template vector for cell therapy, non-viral gene therapy vector, raw material for adeno-associated virus (AAV) or lentiviral vector production, DNA vaccine production, and CAR-T/CAR-NK cell engineering.

Brand names
Nanoplasmid
Other names
Next-generation plasmid vectorMIP vector (minicircle-inspired plasmid)

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