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Clinical trial intelligence report
Phase 1, Open Label Study of CRISPR-CAR Genome Edited T Cells (PBLTT52CAR19) in Relapsed /Refractory B Cell Acute Lymphoblastic Leukaemia
Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.
Generated
Jun 13, 2026
NCT ID
NCT04557436
Status
COMPLETED
Phase
Phase 1
Sponsor
Great Ormond Street Hospital for Children NHS Foundation Trust
Executive brief
Investment-Ready Snapshot
The PBLTT52CAR19 asset, developed by Great Ormond Street Hospital for Children NHS Foundation Trust, targets relapsed/refractory B-cell acute lymphoblastic leukaemia (B-ALL) in pediatric patients. Given the high unmet need in this patient population, the potential for PBLTT52CAR19 to secure molecular remission prior to allogeneic stem cell transplantation positions it favorably in the CAR-T therapy market. The asset's innovative use of CRISPR technology for genome editing may provide a competitive edge over traditional CAR-T therapies, particularly in terms of safety and efficacy. The completion of this Phase 1 trial will be critical for advancing to larger studies and attracting potential partnerships or acquisitions from larger biopharma entities focused on oncology.
Source & freshness
Provenance
https://clinicaltrials.gov/study/NCT04557436
Indication
B Acute Lymphoblastic Leukemia
Modality
gene therapy
Target
CD19+ B cells, utilizing CRISPR-CAR genome editing to modify T cells for enhanced anti-leukemic activity.
Intervention
PBLTT52CAR19
Source record
Protocol Description
Detailed source ingestion pending.
Source record
Outcome Measures
Detailed source ingestion pending.
Source record
Eligibility
Detailed source ingestion pending.
AI analysis
Known Results And Readout Context
Detailed source ingestion pending.
IP intelligence
Patent And IP Landscape
Detailed source ingestion pending.
Source record
Contacts
Detailed source ingestion pending.