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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

Silence Genes

siRNA and other non-coding RNA

Express Genes

Express Genes mRNA

Messenger RNA

Express Genes

Express Genes DNA

DNA

Edit Genes

Edit Genes CRISPR

CRISPR and other gene editing tools

 

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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

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
siRNA, mRNA, DNA, CRISPR
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

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

 


NanoMedU

Precision NanoSystems' learning platform for topics, techniques and technologies to help accelerate non-viral genetic medicine development.

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