• Pioneering
    Gene Therapies

    For Nucleotide Expansion Disorders
    Contact Us
  • ASRE
    Technology

    Destroying Disease causing RNA
    Contact Us
  • Targeting
    Disease

    MYOTONIC DYSTROPHY, HUNTINGTON DISEASE AND 20+ OTHER RELATED DISORDERS
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Our Company

Gene Therapy by Enzerna Biosciences Inc.

Enzerna Biosciences, Inc is a pre-clinical stage company that is leveraging its proprietary RNA editing technology to develop long-term curative gene therapies for rare genetic disorders.

Our Target indications

The Current Problem of Nucleotide Expansion Disorders

Nucleotide expansion disorders, such as Myotonic Dystrophy and Huntington’s Disease, are genetic diseases that typically lead to progressive tissue loss. Life expectancy is about 20 years after clinical diagnosis. Unfortunately, there are no disease modifying therapies.

Patients with nucleotide expansion disorders inherit a gene that is too long – a gene that carries an expansion of a 3-6 nucleotide genetic element that leads to the production of a toxic RNA and/or protein that leads to progressive cell death.

Blood cells for gene therapy research.

Our technology

Enzerna's Gene Therapy Solution

Our solution is to destroy the toxic RNA molecule that is the root cause of nucleotide expansion disorders using Artificial Site-Specific RNA Endonucleases (ASREs). ASREs are rationally designed proteins, that when expressed in cells, can target the expanded repeat array and destroy the disease-causing RNA.

Enzerna has secured an exclusive license for ASRE technology (US Patent No. 9,499,805) from the University of North Carolina-Chapel Hill for all commercial applications.

Gene therapy test tubes in a lab.

“Enzerna's ASRE technology specifically targets disease-causing nucleotide repeats at the RNA level, a specialty that sets it apart from CRISPR/Cas and other DNA modifying technologies ”

— ZEFENG WANG, PHD – SCIENTIFIC FOUNDER

01

Science

A look at the science behind Enzerna’s unique technology and how it effects disease.

02

Myotonic Dystrophy

This type of muscular dystrophy is a genetic disorder that affects muscle function. 

03

Huntington's Disease

Learn about how our therapeutic strategies can target Huntington’s Disease.

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