TECHNOLOGY
Vector-based delivery of “nuclease-free” epigenetic editing
To address the significant therapeutic limitations of current epigenetic editing technologies, nAAVigen Therapeutics has developed a novel strategy utilizing vector-based delivery of non-nuclease gene editing. The company’s proprietary platform is designed to provide a safe and effective therapeutic solution for the most common genetic diseases.
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Refined epigenetic editing mechanism
nAAVigen’s epigenetic editing mechanism has been engineered to be “nuclease-free.” The zinc finger mechanism is not derived from bacteria and enables highly precise, selective epigenetic editing without the risk of producing single or double strand breaks in the DNA. Compared with current technologies, nAAvigen’s mechanism carries lower immunogenic risk and greater specificity to target cells.
IMPROVED VECTOR DELIVERY
Following the identification of AAV6 as the most suitable variant for high-efficiency transduction of primary human hematopoietic stem cells more than a decade ago, nAAVigen has developed capsid-modified NextGen AAV6 vectors, and genome-modified AAV6-B19 hybrid vectors, to produce a best-in-class delivery system for improved transduction at significantly lower doses as well as erythroid lineage-restricted expression. Combined with nuclease-free epigenetic editing, nAAVigen’s NextGen AAV6-B19 hybrid vectors enable a safer and more accessible treatment of human
β-thalassemia and sickle cell disease.
Curing the most common genetic diseases
An estimated 15 million people worldwide are affected by β-thalassemia and sickle cell disease, making them the most common genetic diseases. Epigenetic editing offers hope to patients with these life-threatening diseases, however current treatment requires intensive conditioning and hematopoietic stem cell transplantation.
nAAVigen’s vector-based solution offers an alternative treatment that is safer. In preclinical studies, nuclease-free epigenetic editing successfully reactivates the production of fetal hemoglobin in the cells of patients with sickle cell disease. Additional research indicates that the company’s novel platform may have application in other hematologic and genetic diseases.