Delving into the Latest Updates on HBA1 modified CD34+ HSPCs with Synapse

Overview

Basic Info

Drug Type

Stem cell therapy

Synonyms-

Target

HBA1

Action

inhibitors

Mechanism

HBA1 inhibitors(hemoglobin subunit alpha 1 inhibitors)

Therapeutic Areas

Congenital DisordersHemic and Lymphatic DiseasesOther Diseases

Active Indication-

Inactive Indication

Beta-Thalassemia

Originator Organization

Stanford University

Active Organization-

Inactive Organization

Stanford University

License Organization-

Drug Highest PhasePendingPreclinical

First Approval Date-

Regulation-

Author: Rettig, Garrett R ; Sheehan, Vivien A ; Bode, Nicole M ; Dever, Daniel P ; Kurgan, Gavin ; Collingwood, Michael A ; Uchida, Naoya ; Camarena, Joab ; Ponce, Ezequiel ; Porteus, Matthew H ; Zhao, Feifei ; Bak, Rasmus O ; Majeti, Ravindra ; Cromer, M Kyle ; Martin, Renata M ; Behlke, Mark A ; Lemgart, Viktor T ; Zhang, Yankai ; Goyal, Ankush ; Vakulskas, Christopher A ; Tisdale, John F ; Srifa, Waracharee ; Lesch, Benjamin J

β-Thalassemia pathology is due not only to loss of β-globin (HBB), but also to erythrotoxic accumulation and aggregation of the β-globin-binding partner, α-globin (HBA1/2). Here we describe a Cas9/AAV6-mediated genome editing strategy that can replace the entire HBA1 gene with a full-length HBB transgene in β-thalassemia-derived hematopoietic stem and progenitor cells (HSPCs), which is sufficient to normalize β-globin:α-globin messenger RNA and protein ratios and restore functional adult hemoglobin tetramers in patient-derived red blood cells. Edited HSPCs were capable of long-term and bilineage hematopoietic reconstitution in mice, establishing proof of concept for replacement of HBA1 with HBB as a novel therapeutic strategy for curing β-thalassemia.

100 Deals associated with HBA1 modified CD34+ HSPCs

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