Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
ALS20aI
- Development stage
- Preclinical
- Lead developer
- Children's Hospital of Philadelphia
- Modality
- Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous (through Hematopoietic Stem Cell Transplantation)
01
Overview
**ALS20aI** is a lentiviral gene therapy vector designed to express human alpha-globin for the treatment of *alpha-thalassemia*. It uses the human alpha-globin gene under the control of the beta-globin promoter and locus control region to achieve high-level, sustained expression of alpha-globin in transplanted hematopoietic stem cells[6][1][4]. Preclinical studies have demonstrated that ALS20aI can correct defective alpha-globin production, support red blood cell survival, and restore hemoglobin synthesis in mouse models of alpha-thalassemia[1][4][23]. The therapy is intended to provide long-lasting correction of genetic defects by modifying the patient's own stem cells and enabling them to produce sufficient alpha-globin[1][6].
- Brand names
- ALS20aIALS-20aIALS 20aI
- Other names
- ALS20aIALS-20aIALS 20aI
02
Targets
DNA
Beyond the preview
Go deeper on ALS20aI.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on ALS20aI.