CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

Inclusion Criteria

• •Diagnosis of sickle cell disease with the homozygous HbS homozygous genotype (HbSS) or an HbSβ thalassemia variant (ie, HbSβ0 thalassemia or HbSβ+ thalassemia) genotype, confirmed by hemoglobin studies.
• •Fetal hemoglobin (HbF) ≤ 15%.
• •Severe sickle cell disease symptomatology, defined as any one or more of the following:
• •1. ≥ 2 episodes of acute chest syndrome in the last 2 years.
• •2. ≥ 3 episodes of severe pain events requiring a visit to a medical facility and treatment with opioids in the last 2 years.
• •3. \> 2 episodes of recurrent priapism in the last 2 years.
• •4. Red-cell alloimmunization (\> 2 antibodies) during long-term transfusion therapy (lifetime history).
• •5. Chronic transfusions for primary or secondary prophylaxis (lifetime history).
• •6. Trans-thoracic echocardiograph evidence of tricuspid valve regurgitant jet velocity ≥ 2.7 m/sec (lifetime history).
• •7. Clinically significant neurologic event (eg, ischemic stroke) or any neurological deficit lasting \> 24 hours.