Compositions and methods for treating beta-hemoglobinopathies

US10961537B2 · kind B2 · utility

Assignees

• CSL Behring Gene Therapy, Inc. · US
• CSL Gene Therapy Pty Ltd. · AU

Inventors

• Jeffrey D. Ahlers · Winterville, US
• Jeffrey S. Bartlett · Columbus, US
• Chi-Lin Lee · Jinshanmian, TW
• Gene-Errol Eugenio RINGPIS · West Hollywood, US
• Geoffrey Phillip Symonds · Mandurah, AU
• Ming Yan · Los Angeles, US

Key dates

• Filing date: Jul 17, 2018
• Grant date: Mar 30, 2021
• Expiry date: Jul 17, 2038

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2830/008
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or β-thalassemia or to reduce symptoms thereof.