Mandy Boontanrart - Ariya Bio

B-Hemoglobinopathies are one of the most common genetic diseases worldwide with still no curative therapy other than allogeneic stem cell transplant. To address this, we are developing a CRISPR/Cas9 therapeutic approach to reactivate fetal hemoglobin that will ameliorate the disease symptoms. The genetic region we are targeting has not been proposed or explored for therapeutic benefit before.