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

RareDisease2

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.

Learn More
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.
MENU
X

Get Started

To learn how Precision NanoSystems accelerates nanomedicine development from an idea to clinical applications, contact our Technical Sales Team.

Get in Touch

Rare Disease Resources

Publication - Abstract

July 16, 2015

Journal of Physical Chemistry

Microfluidic Mixing: A General Method for Encapsulating Macromolecules in Lipid Nanoparticle Systems

Leung AK, Tam YY, Chen S, Hafez IM, Cullis PR

Read More

Webinar

November 22, 2016

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

Read More

Publication - Abstract

December 28, 2014

Journal of Controlled Release

Development of Lipid Nanoparticle Formulations of siRNA for Hepatocyte Gene Silencing Following Subcutaneous Administration

Chen S, Tam YY, Lin PJ, Leung AK, Tam YK, Cullis PR

Read More

Video

November 20, 2016

"Nanoparticles: Application of the Technology at Roche" - Kirsten Gräser

Read More

Publication - Abstract

August 14, 2012

Molecular Therapy: Nucleic Acids

Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA

Belliveau NM, Huft J, Lin PJ, Chen S, Leung AK, Leaver TJ, Wild AW, Lee JB, Taylor RJ, Tam YK, Hansen CL, Cullis PR

Read More

Poster

July 01, 2018

Robust low-volume production of nanoparticles for genetic manipulation of cells

Read More PDF
Resource Center