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NCT04443907TERMINATEDanonymous

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

Sponsor

Source record

Novartis Pharmaceuticals

Phase

Source record

Phase 1/2

Modality

AI-normalized

protein therapy

Target

AI-normalized

BCL11A

Indication / condition

AI-normalized

Sickle Cell Disease

Intervention

Source record

OTQ923, OTQ923

Source & freshness

Source record

NCT ID

NCT04443907

Original source

ClinicalTrials.gov

Source last updated

Jan 08, 2026

Ingested at

Jun 18, 2026

Internal sync

Jun 18, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

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NCT ID

NCT04443907

Title

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

Sponsor

Novartis Pharmaceuticals

Status

TERMINATED

Phase

Phase 1/2

Condition raw

Sickle Cell Disease

Condition normalized

Sickle Cell Disease

Modality raw

protein therapy

Modality normalized

protein therapy

Target raw

BCL11A

Target normalized

BCL11A

Interventions

OTQ923, OTQ923

Public preview

Source record

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.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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