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.
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.
“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
A look at the science behind Enzerna’s unique technology and how it effects disease.
This type of muscular dystrophy is a genetic disorder that affects muscle function.
Learn about how our therapeutic strategies can target Huntington’s Disease.