Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
autologous CD34+ hematopoietic stem cells (lentiviral ADA gene-modified)
- Development stage
- Unknown
- Lead developer
- University of California, Los Angeles
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
This autologous ex vivo gene therapy is designed for the treatment of adenosine deaminase (ADA) deficiency severe combined immunodeficiency (ADA-SCID). The process involves harvesting CD34+ hematopoietic stem cells from the patient and transducing them with a lentiviral vector carrying a functional copy of the human ADA gene. These modified cells are then re-infused into the patient, typically following a conditioning regimen. The therapy aims to provide a permanent source of ADA-producing cells, thereby restoring immune system development and function. This approach is being investigated by Beijing Children's Hospital as a potentially safer and more effective alternative to gammaretroviral vectors or lifelong enzyme replacement therapy.
- Other names
- ADA-Lentiviral Gene TherapyLentiviral ADA-modified CD34+ cells
02
Targets
ADA (Adenosine deaminase)
Beyond the preview
Go deeper on autologous CD34+ hematopoietic stem cells (lentiviral ADA gene-modified).
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 autologous CD34+ hematopoietic stem cells (lentiviral ADA gene-modified).