TrialSignal
Clinical trial intelligence report
A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease
Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.
Generated
Jun 18, 2026
NCT ID
NCT04443907
Status
TERMINATED
Phase
Phase 1/2
Sponsor
Novartis Pharmaceuticals
Executive brief
Investment-Ready Snapshot
The clinical trial for OTQ923, a genome-edited autologous hematopoietic stem and progenitor cell therapy targeting BCL11A to increase fetal hemoglobin levels, was terminated early due to business reasons, indicating potential strategic shifts within Novartis Pharmaceuticals. The market for sickle cell disease therapies is expanding, particularly with the rise of gene therapies. However, the competitive landscape includes established therapies and emerging gene-editing technologies, necessitating rigorous diligence to assess Novartis's positioning and future strategies in this space.
Source & freshness
Provenance
https://clinicaltrials.gov/study/NCT04443907
Indication
Sickle Cell Disease
Modality
protein therapy
Target
BCL11A
Intervention
OTQ923, OTQ923
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.