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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 - Summary

December 19, 2017

Cell Reports

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

An D, Schneller JL, Frassetto A, Liang S, Zhu X, Park JS, Theisen M, Hong SJ,Zhou J, Rajendran R, BeccaLevy B, Howell R,...

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Poster

June 11, 2018

Rapid development and seamless scale-up of genetic nanomedicines

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Webinar

April 26, 2016

Lipid Nanoparticle Delivery of siRNA to Unravel Chronic Pain

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Publication - Abstract

March 01, 2014

Endocrinology

IGFBP2 Is Neither Sufficient nor Necessary for the Physiological Actions of Leptin on Glucose Homeostasis in Male ob/ob Mice

Neumann UH, Chen S, Tam YY, Baker RK, Covey SD, Cullis PR, Kieffer TJ

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Video

August 10, 2017

SAM (Self Amplifying Message) for Rapid Response to Emerging Infectious Diseases - Derek O'Hagan

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Video

July 26, 2016

Microfluidics in Manufacturing of mRNA Nanomedicines | Heinrich Haas, BioNTech

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