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HBB HSC-01

Development stage: Phase 1
Lead developer: Allife Medical Science and Technology
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
Administration: Intravenous

Overview

HBB HSC-01 is a gene therapy developed by Allife Medical Science and Technology Co., Ltd. It utilizes CRISPR/Cas9 technology for gene correction of the HBB gene in patient-specific induced hematopoietic stem cells (iHSCs). This therapy is being investigated for the treatment of β-thalassemia mutations.

Targets

Hb (Hemoglobin)

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