Pioneering Genetic Medicines for Nucleotide Repeat Disorders


Our Idea

The Development of therapeutic Artificial Site-Specific RNA Endonucleases (ASREs), stems from Dr. Zefeng Wang, and his understanding of RNA-Protein interactions. When expressed in cells, ASRE proteins, which cut RNA with high sequence specificity, can be targeted against nucleotide repeats known to cause many disease, including Huntington's disease, and certain forms of ALS. Expression is also reversible, allowing for a high level of control of the ASRE protein. 


“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