(SLE)
BETIBEGLOGEN AUTOTEMCEL(INNC)
AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS OBTAINED FROM MOBILISED PERIPHERAL BLOOD OF PATIENTS WITH BETA-THALASSEMIA, TRANSDUCED EX VIVO WITH BETIBEGLOGENE DAROLENTIVEC (116)(78), A SELF-INACTIVATING HUMAN IMMUNODEFICIENCY VIRUS-1 (HIV-1)-DERIVED LENTIVIRAL VECTOR ENCODING A T87Q-MUTATED FORM OF THE HUMAN HEMOGLOBIN SUBUNIT BETA (HBB, BETA-GLOBIN) GENE UNDER THE CONTROL OF A HUMAN BETA-GLOBIN PROMOTER AND A 3' BETA-GLOBIN ENHANCER(WHO)
DRUG SUBSTANCE (ALSO REFERRED TO AS ACTIVE SUBSTANCE) IN LENTIGLOBIN BB305 DRUG PRODUCT IS AN AUTOLOGOUS CD34+ CELL ENRICHED POPULATION THAT CONTAINS HEMATOPOIETIC STEM CELLS TRANSDUCED WITH LENTIGLOBIN BB305 LENTIVIRAL VECTOR (LVV) ENCODING THE BETA^A-T87Q-GLOBIN GENE. LENTIGLOBIN BB305 DRUG PRODUCT CONSISTS OF THE DRUG SUBSTANCE SUSPENDED IN CRYOPRESERVATION SOLUTION(GB)
AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS TRANSDUCED EX VIVO WITH BETIBEGLOGENE DAROLENTIVEC (116)(78), A SELF-INACTIVATING HUMAN IMMUNODEFICIENCY VIRUS-1 (HIV-1)-DERIVED LENTIVIRAL VECTOR ENCODING A T87Q-MUTATED FORM OF THE HUMAN HEMOGLOBIN SUBUNIT BETA (HBB, BETA-GLOBIN) GENE UNDER THE CONTROL OF A HUMAN BETA-GLOBIN PROMOTER AND A 3' BETA-GLOBIN ENHANCER(WHOX)
GENETICKY MODIFIKOVANÉ AUTOLOGNÍ BUŇKY OBOHACENÉ O CD34+ POPULACI, KTERÁ OBSAHUJE HEMATOPOETICKÉ KMENOVÉ BUŇKY (HSC) TRANSDUKOVANÉ LENTIVIROVÝM VEKTOREM (LVV) KÓDUJÍCÍM BETA^A T87Q-GLOBINOVÝ GEN(CZ)
BETIBEGLOGENUM AUTOTEMCELUM(INNM)