Your Competitive Advantage

Improved in vivo expression, duration, safety, economy and ease-of-use

Smaller backbone

Bigger Yield

The Nanoplasmid™ vector has shown success for both indirect use ( as for viral vectors and transposons manufacturing) and for direct use, as an effective, safe, and economical preferred vector for gene therapy and manufacturing. It performs favorably compared to legacy (antibiotic resistance) plasmids and more modern alternatives (such as minicircles, midges, MIPs and doggy bones) that are either inefficient or that require cumbersome and costly post-purification processing – and/or are not regulatory compliant (antibiotic-free). In addition, the Nanoplasmid’s origin of replication (R6K) has host-restricted replication, compared to traditional plasmids’ promiscuity of replication throughout gram negative hosts. In summary, the Nanoplasmid™ is an ideal non-viral vector for gene therapy of human cells and tissues, offering improved expression and duration of expression, as well as an excellent safety profile, ease and economy of purification. Today, Nanoplasmids are being used by more than 50 gene therapy and immunotherapy organizations worldwide.

  • Higher levels of gene expression than plasmids and minicircles

  •  Greater potency, longer duration 

  • Demonstrated as safe in multiple clinical trials 

  • Low toxicity, in vitro or in vivo

  •  Non-promiscuous, replicates only in the Nanoplasmid E coli host strain 

Nanoplasmids™ Project Development Timeline

Concept

Genes of Interest

Design

Client & NTC

Synthesis

Optimization

Cloning

Nanoplasmids™

Manufacturing

Fermentation/Purification

Nanoplasmids™ can be used directly or indirectly

Versatile Solutions

The Nanoplasmid™ can be used for multiple applications providing endless possibilities

Direct Gene Transfer


Nanoplasmid™ Vaccine Retrofits will:

  • Improve performance: Increase transgene expression level and duration and decrease innate immune activation while increasing immune response by activating imunostimulatory Nanoplasmid™ backbone

  • Reduce regulatory concern: eliminate bacterial backbone encoded marker gene transfer and not replicate outside of the engineered production host strain

  • Improve manufacturing: improve plasmid manufacturing in E. coli


Vector Nanoplasmid™ Retrofits will:

  • Improve performance: Increase transgene expression level and duration and decrease innate immune activation

  • Reduce regulatory concern: eliminate bacterial backbone encoded marker gene transfer and not replicate outside of the engineered production host strain

  • Improve manufacturing: improve plasmid manufacturing in E. coli

Indirect Gene Transfer

Transposon (e.g. PiggyBac, Sleeping Beauty non-viral vectors) will:

  • Improve primary cell therapy transposition rates and lower transfection toxicity (improved performance; e.g. up to six-fold higher yield of CD19-CAR T cells compared to Plasmid vectors

  • Eliminate bacterial backbone encoded antibiotic marker genes (reduced regulatory concern)

  • Not change the sequence or composition of the integrated transposon (reduced regulatory concern)

  • Not replicate outside of the engineered production host strain (reduced regulatory concern)

Gene Editing (e.g. Homology-Directed Repair/CRISPR-Cas9 non-viral vectors) Nanoplasmid™ vectors will:

  • Lower transfection toxicity (improved performance)

  • Eliminate bacterial backbone encoded antibiotic marker genes (reduced regulatory concern)

  • Not change the sequence or composition of the integrated transgene (reduced regulatory concern)

  • Not replicate outside of the engineered production host strain (reduced regulatory concern)

Lentivirus vector Nanoplasmid™ NP Retrofits will:

  • Improve virus particle manufacturing titers (improved performance up to 3-5 fold increase in transducing units)

  • Eliminate bacterial backbone encoded antibiotic marker genes (reduced regulatory concern)

  • Not change the sequence or composition of the therapeutic virus particle (reduced regulatory concern)

  • Not replicate outside of the engineered production host strain (reduced regulatory concern)

AAV vector NP Retrofits will:

  • Eliminate bacterial backbone encoded marker gene transfer (reduced regulatory concern)

  • Improve plasmid manufacturing in E. coli, especially with the ITR containing transfer vector which is difficult to replicate with the pUC origin (improved manufacturing)

  • Not change the sequence or composition of the therapeutic virus particle (reduced regulatory concern)

  • Not replicate outside of the engineered production host strain (reduced regulatory concern)

polyA mRNA vector NP Retrofits will:

  • Improve plasmid manufacturing in E. coli (improved manufacturing)

Licensing

For more information regarding sales or technology licensing, email licensing@natx.com.