Gene Therapy With The Lentiviral Vector Tns9.3.55 Produces Long-Term Improvement In Severe ß-Thalassemia

Author(s): Aurelio Maggio, Michel Sadelain, Isabelle Riviere, Xiuyan Wang, Simona Raso, Santina Acuto, Rita Barone, Rosario Di Maggio, Lorella Pitrolo, Farid Boulad

EHA Library. Raso S. 06/12/20; 293982; EP1497

Abstract: EP1497

Type: e-Poster
Presentation during EHA25: All e-Poster presentations will be made available on the on-demand Virtual Congress platform as of Friday, June 12 at 08:30 CEST and will be accessible until October 15, 2020.

Background

β-thalassemia is the most prevalent monogenic disease for which gene therapy with autologous hematopoietic progenitor cells (HPC) modified to express ß-globin represents a potential therapeutic option. The most relevant clinical variables influencing success of gene therapy in ß-thalassemia are patient genotype, myeloablative conditioning regimen, number and type of progenitor cells, and vector copy number (VCN).

Aims

We report the results of a safety and efficacy study of the use of gene therapy with the human globin TNS9.3.55 vector in three patients at Memorial Sloan Kettering Cancer Center between November 2012 and May 2015, with a follow-up of 5-7 years. The results of 3 patients treated and followed at Department of Hematology and Rare Diseases European Reference Network Center for Rare Hematological Diseases (Hospital Villa Sofia-V. Cervello) are reported here.

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