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

Video

November 30, 2016

“Targeted Lipid/Peptide Nanocomplexes for Nucleic Acid Delivery” - Stephen Hart, UCL

Read More

Publication - Abstract

October 07, 2015

eLife

Precise Let-7 Expression Levels Balance Organ Regeneration against Tumor Suppression

L. Wu, L.H. Nguyen, K. Zhou, T.Y. de Soysa, L. Li, et al.

Read More

Video

July 26, 2016

Microfluidics in Manufacturing of mRNA Nanomedicines | Heinrich Haas, BioNTech

Read More

Publication - Abstract

November 16, 2014

Advances in Genetics

Lipid Nanoparticles for Short Interfering RNA Delivery

Leung AKK, Tam YYC, Cullis PR

Read More

Publication - Abstract

April 18, 2018

Journal of Leukocyte Biology

Lipofection of Plasmid DNA into Human Mast Cell Lines using Lipid Nanoparticles Generated by Microfluidic Mixing

Duguay, B. A., Huang, K. W., & Kulka, M

Read More

Publication - Abstract

September 18, 2013

Pharmaceutics

Advances in Lipid Nanoparticles for siRNA Delivery

Tam YY, Chen S, Cullis PR

Read More
Resource Center