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To learn how Precision NanoSystems accelerates nanomedicine development from an idea to clinical applications, contact our Technical Sales Team.
Get in TouchHundreds 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 200 peer reviewed publications, many of which demonstrate exciting proof of concepts for gene therapies that include:
Numerous pharmaceutical and biotechnology companies have adopted PNI solutions for accelerating their gene and cell therapy programs.
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.
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.
To learn how Precision NanoSystems accelerates nanomedicine development from an idea to clinical applications, contact our Technical Sales Team.
Get in TouchPublication - Abstract
September 13, 2016
Chemical Society Reviews
Publication - Summary
December 03, 2013
Molecular Therapy—Nucleic Acids
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September 24, 2017
Nano Letters
Publication - Summary
July 13, 2017
Cell
Publication - Abstract
June 10, 2015
ACS Nano