Exa-cel

BIOLOGICAL2 trials

Sponsors

Vertex Pharmaceuticals Incorporated

Conditions

Hematological DiseasesHemoglobinopathiesSickle Cell Disease

Phase 2

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

CompletedNCT03745287

Vertex Pharmaceuticals IncorporatedHematological Diseases, Hemoglobinopathies, Sickle Cell Disease

Start: 2018-11-27End: 2025-07-07Updated: 2026-09-16

Phase 3

Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype

WithdrawnNCT05951205

Vertex Pharmaceuticals IncorporatedSickle Cell Disease

Start: 2027-07-31End: 2033-12-31Updated: 2026-03-25

Related Papers

Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.

2025-12-012 citations

Exagamglogene Autotemcel for Severe Sickle Cell Disease

New England Journal of Medicine2024-04-24294 citations

Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches

International Journal of Molecular Sciences2024-02-2040 citations

5612617 EFFICACY AND SAFETY OF A SINGLE DOSE OF EXAGAMGLOGENE AUTOTEMCEL FOR TRANSFUSION-DEPENDENT-THALASSEMIA AND SEVERE SICKLE CELL DISEASE

HemaSphere2023-04-013 citations

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood2022-11-1520 citations

OS BENEFÍCIOS DA APLICAÇÃO DA TERAPIA GÊNICA CRISPR/CAS9 NA ANEMIA FALCIFORME

Hematology Transfusion and Cell Therapy2022-10-01

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

New England Journal of Medicine2020-12-051721 citations

Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells

Blood2020-11-0449 citations

Treating sickle cell anemia: A new era dawns

American Journal of Hematology2020-01-1124 citations