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Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy
- Source :
- Blood; December 2017, Vol. 130 Issue: 1, Number 1 Supplement 1 p4609-4609, 1p
- Publication Year :
- 2017
-
Abstract
- Background:LentiGlobin Drug Product (DP) contains autologous CD34+ cells transduced with the betibeglogene darolentivec (BB305) lentiviral vector, encoding a human β-globin gene with a point mutation (AT87Q) that confers anti-sickling properties similar to those with γ-globin. Human proof of concept for LentiGlobin treatment in transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD) has been established in HGB-205 (NCT02151526), the original clinical study of gene therapy for hemoglobinopathies. Here, we provide an update on 5 previously-reported patients (4 TDT, 1 SCD) as well as early data from 2 additional treated patients with SCD.
Details
- Language :
- English
- ISSN :
- 00064971 and 15280020
- Volume :
- 130
- Issue :
- 1, Number 1 Supplement 1
- Database :
- Supplemental Index
- Journal :
- Blood
- Publication Type :
- Periodical
- Accession number :
- ejs56858098
- Full Text :
- https://doi.org/10.1182/blood.V130.Suppl_1.4609.4609