NanoMedU
Precision NanoSystems' learning platform for topics, techniques and technologies to help accelerate non-viral genomic medicine development.
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Genomic Medicines
End-to-end Solutions to Enable Genomic Medicines
Precision NanoSystems provides a full stack of technologies and solutions with leading experience to accelerate the design, development and manufacturing of promising Genomic Medicines.
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Disease TargetBiology insights can identify target gene(s) driving disease
Genomic Medicine Toolkit
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Genomic Payload PlatformChoose RNA/DNA to silence, express, or edit target gene(s) -
GenVoy™ Delivery PlatformLipid nanoparticles (LNPs) protect & deliver RNA/DNA to target cells -
NanoAssemblr® Manufacturing PlatformMicrofluidics manufactures genomic medicines seamlessly from discovery to clinic
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Drug Development ExpertiseLeverage world-leading expertise in genomic medicine development
Genomic medicines are revolutionizing how diseases are treated by addressing dysfunctional genes and gene networks responsible for a wide range of diseases. Genomic medicines come in several forms that can modulate gene expression or edit genes.
Silence Genes
siRNA and other non-coding RNA
Express Genes
Messenger RNA
Express Genes
DNA
Edit Genes
CRISPR and other gene editing tools
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To learn how Precision NanoSystems can help accelerate the design, development and manufacturing of your Genomic Medicines, contact a Scientific Specialist.
How to Develop a Genomic Medicine
Precision NanoSystems Framework provides a roadmap for genomic medicine development. The specific Workflows for development of vaccines, gene therapy and cell therapy are designed to accelerate genomic medicine development by using the Framework together with components of the Genomic Medicine Toolkit to increase effectiveness and efficiency across the drug development process.
Precision NanoSystems Genomic Medicine Development Framework
Validate molecular target(s) in vitro/vivo
Screen sequence, base modifications and excipient library
Systematically test process and formulation parameters to physico-chemical properties and in vivo biological activity
Down-select lead formulations and optimize key process parameters
Additional animal studies in second species to assess efficacy and toxicity
Process and formulation parameters & analytical methods to ensure product quality and performance are developed and finalized
GMP equipment, manufacturing process, analytical methods and QMS transferred into Clean Room Facility and validated with engineering run.
Investigational New Drug (IND) application submitted
GMP batch production of Nanomedicine for clinical testing - Phase 1, 2 & 3
Instrument, process and analytical methods transferred to commercial production site
New Drug Application (NDA) submitted
Commercial production of Nanomedicine initiated
mRNA & DNA-based vaccines Prophylactic & therapeutic
Infectious Disease & Oncology
Population-based to individualized
Infectious Disease & Oncology
Population-based to individualized
siRNA, mRNA, DNA, CRISPR
Silence, express, or edit genes
Focus on rare disease
Population-based to individualized
Silence, express, or edit genes
Focus on rare disease
Population-based to individualized
siRNA, mRNA, DNA, CRISPR
Immune cells including T-Cells
Focus on oncology
Autologous & allogeneic
Immune cells including T-Cells
Focus on oncology
Autologous & allogeneic