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RNA Platform
As part of Precision Nanosystems' Genetic Medicine Toolkit, we provide partners access to RNA technology to advance genetic medicines that are revolutionizing treatment of cancer, rare disease and infectious disease.
How Genetic Medicine Works

Genetic medicines deliver nucleic acids that instruct cells to express, silence or modify a gene. Genetic medicines exploit pre-existing cellular processes to correct or reverse genetic abnormalities to treat diseases that were previously thought to be "undruggable" such as some rare diseases, cancers and infectious diseases.

 

RNA Platform Image

 

Learn More About Genetic Medicine


Examples of Genetic Payloads

 

 

Silence Genes
V3siRNA

Small interfering RNA (siRNA)

 

regulate gene expression by turning genes "off"

Express Genes
V3mRNA

Messenger RNA (mRNA) 

 

encodes proteins

 

Express Genes
V3DNA

DNA

 

 

transcribed to RNA which is translated to protein

Edit Genes
V3CRISPR

CRISPR and Other Gene Editing Modalities

 

RNA or DNA mediate expression of gene editing components


The Advantage of mRNA

 

RNA medicine does not require nuclear delivery and transcription, making mRNA a simple solution for gene therapy.

 

The effects of mRNA are transient which is inherently safer than episomal systems, or those that integrate into the genome.

 

mRNA does not alter the gene itself but instead regulates expression.

 

An mRNA COVID-19 vaccines from Pfizer/BioNTech has recently received Emergency Use Authorization from the FDA.


 

 

 

 

 

 

RNA Molecule

 

Precision Nanosystems has assembled a team around the self-amplifying mRNA (saRNA) technology platform.


Types of mRNA

 

 

Unmodified mRNA

 

Unmodified mRNA

 

Elicits an immune response, ideal for immunotherapy applications

Base-modified mRNA

 

Modified mRNA

 

Modified mRNA that avoids immune response allowing for broader therapeutic applications

Self-amplifying mRNA

 

Self-Amplifying mRNA

 

Encodes not only a protein but also the non-structural proteins that allow mRNA replication


What is Self-Amplifying mRNA?

 

Self-amplifying mRNA (saRNA) are a subclass of mRNA that that can replicate to produce more copies of mRNA by co-opting the cellular machinery.

 

Learn more by watching saRNA pioneer Dr. Andrew Geall's 2020 webinar. 

 

 

Watch the Full Webinar


saRNA Vaccine Partnerships

Precision Nanosystems has expertise using mRNA vector designs to development saRNA constructs.

Contact Us to Learn More
The Advantages of a Lower Dose

Self-amplifying mRNA contain elements of the alphavirus genome which can mediate RNA replication in the cytosol. This replication allows for amplified antigen production while using a low mRNA dose.


SAM Diagram

saRNA Vaccines Proof of Concept

Dr. Andy Geall's 2012 seminal paper demonstrating saRNA vaccines.

This paper shows that saRNA vaccines elicit immunogenic effects and provides proof-of-concept that a saRNA vaccine platform could address future pandemics and public health crises in a swift and effective manner. 

 

 

 

 

2012 SAM Andy Geall
saRNA Vaccine Case Study
2020 Paper from Dr. Yvonne Perrie, University of Strathclyde.
In this study, several formulations of a saRNA vaccine for rabies were tested, and the lipid nanoparticle formulation proved to have comparable results to an approved rabies vaccine.
SAM CD4 Perrie

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Resource Center

Publication - Abstract

July 01, 2020

Journal of Controlled Release

Delivery of Self-amplifying mRNA Vaccines by Cationic Lipid Nanoparticles: The Impact of Cationic Lipid Select...

G. Lou, G. Anderluzzi, S.T. Schmidt, S. Woods, S. Gallorini, M. Brazzoli, F. Giusti, I. F...

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Publication - Abstract

May 08, 2020

Vaccines

Investigating the Impact of Delivery System Design on the Efficacy of Self-Amplifying RNA Vaccines

G. Anderluzzi, G. Lou, S. Gallorini, M. Brazzoli, R. Johnson, D.T. O'Hagan, B.C. Baudner a...

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Publication - Abstract

April 02, 2020

Journal of Controlled Release

Design of a Novel Vaccine Nanotechnology-based Delivery System Comprising CpGODN-protein Conjugate Anchored to...

D. Chatzikleanthous, S.T. Schmidt, G. Buffi, I. Paciello, R. Cunliffe, F. Carboni, M.R. Ro...

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Publication - Abstract

December 31, 2019

Vaccines

Investigating Prime-Pull Vaccination through a Combination of Parenteral Vaccination and Intranasal Boosting

C.B. Roces, M.T. Hussain, S.T. Schmidt, D. Christensen and Y. Perrie

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Publication - Abstract

May 31, 2019

Vaccine

mRNA Vaccines Against H10N8 and H7N9 Influenza Viruses of Pandemic Potential Are Immunogenic and Well Tolerate...

R.A. Feldman, R. Fuhr, I. Smolenov, A. Ribeiro, L. Panther, M. Watson, J.J. Senn, M. Smith...

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Publication - Summary

May 18, 2019

Annals of Hematology

Lipid Nanoparticle-mediated siRNA Delivery for Safe Targeting of Human CML In Vivo

N. Jyotsana, A. Sharma, A. Chaturvedi, M. Heuser et al.

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