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Full notification Final report - General information Notification Number B/DE/22/PEI4827 Member State to which the notification was sent Germany Date of acknowledgement from the Member State Competent Authority 08/03/2022 Title of the Project Clinical study VX21-CTX001-161 titled: “A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with TransfusionDependent β-Thalassemia or Severe Sickle Cell Disease Proposed period of release: 01/01/1970 to 01/01/1970 Name of the Institute(s) or Company(ies) Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA 02210, USA
Is the same GMO been notified elsewhere by the same notifier? No Has the same GMO been notified elsewhere by the same notifier? Yes If yes, notification number(s): B/DE/18/PEI3279 (CTX001-111); B/DE/19/PEI3506 (CTX001-121); GMO characterization GMO is a: Other Identity of the GMO: The GMO/IMP (CTX001) consists of subject autologous peripheral blood-derived CD34+ human hematopoietic stem and progenitor cells (hHSPCs) modified ex vivo using the CRISPR-Cas9 gene editing technology. The CRISPR-Cas9 editing components are transiently introduced into the target cell population by electroporation as a ribonucleoprotein complex consisting of Cas9 and a gRNA that targets the erythroid lineage-specific enhancer of the BCL11A gene. This process does not use plasmid or viral vectors. The CRISPR-Cas9 gene editing technology is intended to disrupt the erythroid lineage-specific enhancer of the BCL11A gene on chromosome 2 in a site-specific and permanent fashion in order to reactivate transcription and expression of γ-globin, resulting in increased levels of fetal hemoglobin (HbF) for the treatment of β-thalassemia and sickle cell disease. The cells will be used only for therapeutic purposes in the same subject from whom the cells were obtained (autologous application). Information relating to the recipient or parental organisms from which the GMO is derived Common Name: Human Genus: Homo Species: Homo Sapiens Subspecies: Strain: CD34+ Autologous Hematopoietic Stem Cells Pathovar: European Commission administrative Information Consent given by the Member State Competent Authority: Not known
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