Rare Diseases and Unmet Medical Needs

Finding and Treating the Molecular Root Cause

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 200 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.

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.

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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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.

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

A Glu-urea-Lys Ligand-Conjugated Lipid Nanoparticle/siRNA System Inhibits Androgen Receptor Expression in Vivo

Nanocarriers Tryst with the Lysosomes: Implications in Intracellular Drug Delivery and Disease

SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency

Lipid Nanoparticles Deliver Nucleic Acids, Including CRISPR Components, in Primary Neurons

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

Microfluidic-Based Manufacture of siRNA-Lipid Nanoparticles for Therapeutic Applications