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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.
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
End-to-end Solutions to Enable Genetic Medicines