top of page

Developing safer solutions for nuclease-free epigenetic editing
nAAVigen’s vector-based solutions provide a safer delivery system for epigenetic editing and potentially more accessible treatment for diseases like β-thalassemia and sickle cell disease.

Advancing vector-based editing treatments for genetic diseases
Our Platform

β-thalassemia and sickle cell disease
The most common genetic diseases
.png)
β-thalassemia and sickle cell disease
1 out of 600 people
β-thalassemia and sickle cell disease affect 1 out of 600 people, together the most common genetic diseases. While current treatments offer hope for curing these life-threatening diseases, an intensive gene editing process – outside the body followed by hematopoietic stem cell transplantation – coupled with long-term safety concerns, significantly limits therapeutic use.
bottom of page
