8:24 am Bluebirdbio sells off after updating data abstracts at ASH; two B0/B0 genotypes needed a blood transfusion, one remains independent -- call at 8:30 ( BLUE) :
"We are pleased to see a median HbAT87Q level of 5.2 g/dL in the seven Northstar patients with beta-thalassemia major of all genotypes followed for six months or more, as this represents a substantial proportion of their total hemoglobin. It is exciting that transfusion independence has been achieved in all of our patients with beta-thalassemia major with non-0/0 genotypes followed for at least six months in Northstar and HGB-205. Varying degrees of transfusion reduction have been observed in patients with 0/0 genotypes. As would be expected, longer follow-up is required to assess the extent of HbAT87Q production and the impact on transfusion requirements in these patients with 0/0 genotypes since they produce no functional beta-globin at baseline. Turning to sickle cell disease, our first treated patient is producing increased levels of HbAT87Q since we last reported on this study in June. HbAT87Q represented 48 percent of total hemoglobin at nine months post-infusion, and the patient remains transfusion independent without sickle cell-related adverse events as of the data cut-off. These results support the transformative potential of gene therapy, and we look forward to sharing more data at ASH." HGB-204 Abstract 201: All four non-0/0 subjects have been transfusion-free for at least 90 days, with a median of 287 days transfusion-free (range: 171 to 396 days).
Two of the 0/0 subjects have received a single transfusion post-discharge, and one remains transfusion-dependent. Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector (Abstract #202) The subject with severe SCD is producing ~ 51.5% anti-sickling hemoglobin (48 percent HbAT87Q, 1.8 percent HbF, 1.7 percent HbA2) at nine months post-infusion. The subject with severe SCD remains free of transfusions. The subject with severe SCD has not had a post-treatment hospitalization for a disease-related event despite ceasing chronic transfusions on Day +88. Both subjects with beta-thalassemia major have remained transfusion-free for at least 15 months post-infusion, with consistent expression of HbAT87Q -- both subjects are 0/E genotype |
