Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
CTLA4 gene-edited T cells
- Development stage
- Preclinical
- Lead developer
- University College London
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Cell Therapies
- Administration
- Intravenous
01
Overview
CTLA4 gene-edited T cells is an autologous, ex vivo gene-edited T-cell therapy being developed by University College London (UCL) in collaboration with Great Ormond Street Hospital (GOSH) and NHS Blood and Transplant (NHSBT), with funding from LifeArc. The therapy is designed to treat CTLA-4 insufficiency, a rare and life-threatening inborn error of immunity. Using CRISPR/Cas9 gene-editing technology and an adeno-associated virus serotype 6 (AAV6) donor template, the therapy precisely inserts a corrected CTLA-4 cDNA into the first intron of the endogenous CTLA-4 genomic locus in a patient's own CD4+ T cells. This homology-directed repair (HDR) approach preserves native regulatory elements, allowing for physiological, regulated expression of the CTLA-4 protein. Preclinical studies have demonstrated restoration of CTLA-4 expression and rescue of CD80 and CD86 transendocytosis, with a Phase I clinical trial planned to begin in 2028.
- Other names
- CTLA-4 gene-edited T cellsCTLA4 gene-edited T cellsCTLA 4 gene-edited T cellsautologous CTLA-4 gene-edited T cells
Beyond the preview
Go deeper on CTLA4 gene-edited T cells.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect 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 CTLA4 gene-edited T cells.