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

About the study

This study is evaluating a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product – OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Study point of contact

Novartis Pharmaceuticals
[email protected]
Novartis Pharmaceuticals


2 Years - 40 Years




Phase 1/Phase 2

Study type






participation requirements

Male or female subjects age 2-40 years inclusive
Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC, HbS/β0-thalassemia or others)
Performance status >70% (Karnofsky for subjects >16 years of age and Lansky for subjects <16 years of age) At least one of the following indicators of disease severity as defined in the protocol - Vaso-occlusive pain crisis, Acute chest syndrome, Recurrent priapism, prior stroke, receive chronic transfusions, Red cell alloimmunization Subjects, who have failed, not tolerated or refused hydroxyurea therapy.

participation restrictions

Available matched related donor for HSCT
Clinically significant active infection
Seropositive for HIV or HTLV
Active known malignancy, myelodysplasia, abnormal cytogenetics or immunodeficiency
Prior HSCT or gene therapy
Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis
Protocol defined iron overload
Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya
Severe or progressive arteriopathy or cerebrovascular disease, including Moyamoya

Other protocol defined inclusion/exclusion criteria may apply

Last updated 2023-01-09