Mandy Boontanrart - Ariya Bio
Reactivating fetal hemoglobin in B-Hemoglobinopathy patients
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.
Mandy Boontanrart