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ALS20a
- Development stage
- Preclinical
- Lead developer
- Children's Hospital of Philadelphia
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
ALS20a (also written as ALS20α) is an investigational lentiviral vector-based gene therapy designed for the treatment of alpha-thalassemia, including HbH disease. Developed by researchers at the Children's Hospital of Philadelphia (CHOP), ALS20a delivers a functional human alpha-globin gene under the control of the beta-globin promoter and its locus control region (LCR). By introducing a functional copy of the alpha-globin gene into autologous hematopoietic stem and progenitor cells (HSPCs), ALS20a aims to restore the balance of alpha- and beta-globin chains, reduce the formation of nonfunctional beta-globin tetramers (HbH), and correct the disease phenotype in patients with severe alpha-thalassemia.
- Other names
- ALS20-alphaALS-20-alphaALS 20-alphaALS20alphaALS-20alphaALS 20alpha
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