NanoMedU
Precision NanoSystems' learning platform for topics, techniques and technologies to help accelerate non-viral genetic medicine development.
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Genetic Medicines
Genetic medicines are revolutionizing how diseases are treated by addressing dysfunctional genes and gene networks responsible for a wide range of diseases. Genetic 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
How to Develop a Genetic Medicine
PNI’s Framework provides a roadmap for genetic medicine development. The specific Workflows for development of vaccines, gene therapy and cell therapy are designed to accelerate genetic medicine development by using the Framework together with components of the Genetic Medicine Toolkit to increase effectiveness and efficiency across the drug development process.
PNI’s Genetic 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
End-to-end Solutions to Enable Genetic Medicines
Precision NanoSystems provides a full stack of technologies and solutions with leading experience to accelerate the design, development and manufacturing of promising Genetic Medicines.
Genetic Medicine Toolkit
Essential technologies to facilitate development of Genetic Medicines
