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Areas of Interest
Rare Diseases
Finding and Treating the Molecular Root Cause

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Hundreds of millions of people are affected by over 6000 rare diseases. Most of these diseases are genetic in origin. Gene-based medicines can address these diseases at their molecular cause rather than treating symptoms. Precision NanoSystems is uniquely suited to offer a suite of solutions to help drug developers accelerate their genetic medicine programs through advanced technology and expertise in formulating and manufacturing genetic medicines.

 

Our NanoAssemblr technology has been featured in over 100 peer reviewed publications, many of which demonstrate exciting proof of concepts for gene therapies that include:

 

  • RNA interference (RNAi) to silence disease causing genes
  • Messenger RNA therapy for protein or enzyme replacement
  • CRISPR Cas9 and other gene editing approaches to more permanently correct genes

 

Numerous pharmaceutical and biotechnology companies have adopted PNI solutions for accelerating their gene and cell therapy programs. 


Examples

NanoAssemblr Technology is used by innovative research institutes, top pharmaceutical companies and disruptive biotech companies to develop gene-based therapies for rare genetic diseases and other diseases with unmet medical need.





Treating Loss-of-Function Mutations with Messenger RNA

 

 

Methylmalonic Acidemia is a rare metabolic disease caused by a genetic mutation affecting an enzyme essential for digesting certain amino acids. The loss of enzyme function leads to brain disease and developmental delays. Moderna Therapeutics, an mRNA therapeutics company have published a proof of concept for restoring enzyme function. Their approach involved encoding the enzyme in an mRNA, encapsulating it into an LNP using NanoAssemblr technology and delivering the formulation systemically. Functional proteins are then produced in the liver, thus restoring metabolic function while demonstrating impressive safety.

 

Read more.

An et al




CRISPR/Cas9 Genome Editing for Lasting Treatment of Amyloid Disease

 

Hereditary transthyretin amyloidosis is a rare disease where a mutation in the protein transthyretin (TTR)causes it to misfold into amyloid plaques that present a wide range of symptoms, mostly neurological. Intellia Therapeutics, a biotech focused on gene editing treatments, reported a single treatment with CRISPR components resulted in over 97% knockdown of TTR that lasted at least 12 months. They used NanoAssemblr technology to simultaneously package both mRNA encoding the Cas9 endonuclease and sgRNAs targeting TTR into lipid nanoparticles (LNPs). These advances offer a promising glimpse into the lasting power of gene editing approaches for treating inherited diseases.

 

Read more.

2018 Finn et al

Rapid, efficient development of genetic medicines
Genetic medicines allow treatment of genetic diseases at their molecular root cause, and lipid nanoparticles are at the forefront of delivering gene therapies.

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Nucleic Acid Lipid Nanoparticle LNP
Manufacturing Technology
NanoAssemblr technology is trusted by over 70% of top pharmaceutical companies, 80+ innovative biotech companies and 70+ renowned research institutes across all stages of nanomedicine development.
Nanoparticle Technology
Our reagents and transfection kits allow research access to lipid nanoparticle formulations featuring ionizable lipids similar to those in clinically validated formulations. These offer high encapsulation and transfection efficiency with no observed toxicity in numerous primary cell types and in vivo models.
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Rare Disease Resources

Publication - Abstract

December 24, 2016

Molecular Therapy

The Niemann-Pick C1 Inhibitor NP3.47 Enhances Gene Silencing Potency of Lipid Nanoparticles Containing siRNA

Wang H, Tam YY, Chen S, Zaifman J, van der Meel R, Ciufolini MA, Cullis PR

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Publication - Summary

April 27, 2017

Molecular Therapy

Preclinical and Clinical Demonstration of Immunogenicity by mRNA Vaccines against H10N8 and H7N9 Influenza Viruses

Bahl K, Senn JJ, Yuzhakov O, Bulychev A, Brito LA, Hassett KJ, Laska ME, Smith M, Almarsson Ö, Thompson J, Ribeiro AM

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Publication - Summary

September 03, 2015

Nano Letters

An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo

Bin Li, Xiao Luo, Binbin Deng, Junfeng Wang, David W. McComb, Yimin Shi, Karin M. L. Gaensler, Xu Tan, Amy L. Dunn, Bryc...

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Publication - Abstract

September 18, 2013

Pharmaceutics

Advances in Lipid Nanoparticles for siRNA Delivery

Tam YY, Chen S, Cullis PR

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Webinar

November 22, 2016

LUNAR™ Nanoparticles: A Versatile RNA Delivery System | Priya Karmali, Arcturus

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Webinar

March 16, 2017

Targeted DNA Therapeutics - Challenges and Opportunities for Hepatic Diseases

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