Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy

Allogeneic hematopoietic stem cell (HSC) transplantation (allo-HSCT) is a treatment option for several monogenic diseases; however, its use is limited by the need for a matched donor and risk of HSCT-related complications. Autologous HSC gene addition does not have some of these limitations and may have similar efficacy with an improved safety profile. Ex vivo gene addition therapy using lentiviral vectors (LVV) is being evaluated in patients with transfusion-dependent β-thalassemia (TDT) using betibeglogene autotemcel (beti-cel, LentiGlobin for TDT) in the HGB-204, -205, -207, and -212 studies, sickle cell disease (SCD) using LentiGlobin for SCD in HGB-205 and -206, and cerebral adrenoleukodystrophy (CALD) using Lenti-D in ALD-102.

Source: Biology of Blood and Marrow Transplantation - January 24, 2020 Category: Hematology Authors: Mark C. Walters, Franco Locatelli, Adrian J. Thrasher, John F. Tisdale, Paul J. Orchard, Christine N. Duncan, J örn-Sven Kühl, Satiro Nakamura De Oliveira, Martin G. Sauer, Andreas E. Kulozik, Evangelia Yannaki, Suradej Hongeng, Markus Y. Mapara, Lakshm Tags: 48 Source Type: research