Efficacy and Safety in Patients (Pts) with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Lovo-cel gene therapy uses autologous transplantation of hematopoietic stem and progenitor cells (HSPCs) transduced with the BB305 lentiviral vector encoding a modified β-globin gene, which produces an anti-sickling hemoglobin (Hb), HbAT87Q. To report efficacy and safety from HGB-206 (phase 1/2; NCT...

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Published in:Transplantation and cellular therapy 2024-02, Vol.30 (2), p.S230-S231
Main Authors: Kanter, Julie, Thompson, Alexis A., Kwiatkowski, Janet L., Parikh, Suhag, Mapara, Markus Y., Rifkin-Zenenberg, Stacey, Aygun, Banu, Kasow, Kimberly A., Gupta, Ashish O., Zhang, Lixin, Sheldon-Waniga, Emily, Gallagher, Meghan, Gruppioni, Katiana, Chawla, Anjulika, Elliot, Heidi, Pierciey, Francis J., Walters, Mark C., Tisdale, John F.
Format: Article
Language:English
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Summary:Lovo-cel gene therapy uses autologous transplantation of hematopoietic stem and progenitor cells (HSPCs) transduced with the BB305 lentiviral vector encoding a modified β-globin gene, which produces an anti-sickling hemoglobin (Hb), HbAT87Q. To report efficacy and safety from HGB-206 (phase 1/2; NCT02140554) and HGB-210 (phase 3; NCT04293185), including data from pediatric pts (age 12 to
ISSN:2666-6367
2666-6367
DOI:10.1016/j.jtct.2023.12.302