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Nat Med: Long-term outcomes of lentiviral gene therapy for beta-hemoglobinopathies

 Last Update: 2022-02-19
 Source: Internet
 Author: User

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Sickle cell disease and transfusion-dependent beta-thalassemia are the most common monogenic genetic disorders worldwide

Recently, a research article was published in the top medical journal Nature Medicine.

HGB-205 is a Phase 1/2, open-label, single-arm, non-randomized intervention study with a duration of 2 years, conducted at a single center, followed by observation of LTF-303 (NCT02633943) and LTF-307 (NCT02633943) and LTF-307 ( NCT04628585) for transfusion-dependent β-thalassemia and sickle cell disease, respectively

Four patients with transfusion-dependent beta-thalassemia and three patients with sickle cell disease, aged 13-21 years

CONCLUSIONS: All patients with transfusion-dependent β-thalassemia were transfusion-free and had improved erythropoiesis and iron overload

All patients with transfusion-dependent β-thalassemia were transfusion-free and had improved erythropoiesis and iron overload

Original source:

Elisa Magrin, et al.

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

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