Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
NTLA-5001
- Development stage
- Unknown
- Lead developer
- Intellia Therapeutics
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, TCR-Engineered T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
- Administration
- Intravenous
01
Overview
NTLA-5001 is an autologous T cell receptor (TCR)-T cell therapy developed using *ex vivo* CRISPR-Cas9 genome editing. The patient's own T cells are collected, and the endogenous TCR is replaced with a natural, high-avidity TCR (identified from healthy donors) with specificity for the Wilms' Tumor 1 (WT1) antigen, which is overexpressed in acute myeloid leukemia (AML) and other tumor types. The therapy involves CRISPR-Cas9-mediated replacement of the TRAC locus with the WT1 TCR and site-specific knockout of the TRBC locus, further engineered to enhance cell health, safety, and function. CRISPR reagents are delivered via lipid nanoparticles (for gene disruption) and adeno-associated virus vectors (for WT1 TCR transduction). NTLA-5001 is dosed intravenously after lymphodepleting chemotherapy and is being studied for efficacy and safety in AML, particularly in HLA-A*02:01 positive subjects[1][2][3][4][5].
- Brand names
- NTLA-5001NTLA5001NTLA 5001
- Other names
- NTLA-5001NTLA5001NTLA 5001
02
Targets
HLA-A*02 (Human leukocyte antigen A*02 complexed peptide)
Beyond the preview
Go deeper on NTLA-5001.
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 NTLA-5001.