Genetic Correction of Hemoglobinopathy Including Sickle Cell Disease
Summary
A gene therapy method for treating sickle cell disease (SCD) using a modified stem cell that produces corrective antisickling hemoglobin.
Overview
Fetal hemoglobin (HbF) produced during fetal life and the first 6-9 months of age has strong antisickling properties and protects an infant from sickling in the first year of life. However, HbF production is temporary, and the disease arises when defective sickle hemoglobin (HbS) genes take over. Dr. Malik has developed a therapy that achieves genetic correction of autologous bone marrow stem cells using a lentiviral vector encoding a gene that produces gamma-globin at a titer and affinity level that leads to permanent production of fetal hemoglobin, outcompeting production of defective HbS. It offers the possibility of a one-time treatment, resulting in a lifelong correction.
Applications
A therapeutic application for sickle cell disease and B-thalassemias (BT).
Value Proposition
Previous treatment methods required donors or lifelong transfusions. This vector
has no immune side effects, is available to all patients and is a one-time treatment
with lifelong correction of SCD.
Market Overview
Nearly 1,000 babies are born with SCD per day in Africa, and SCD affects over 110,000
Americans. Healthcare costs for US patients with SCD exceeds $1.2 billion.
Investigator
Punam Malik, MD, Division of Experimental Hematology & Cancer Biology
