IMB-2011010
/ Purdue, AbCellera
- LARVOL DELTA
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November 04, 2025
Correction of ineffective erythropoiesis and durable clinical benefit with exagamglogene autotemcel for transfusion-dependent β-thalassemia
(ASH 2025)
- " CLIMB-111 is an ongoing 2-y, Phase 3 trial of exa-cel in TDT pts aged 12-35 y. The primaryendpoint is transfusion independence defined as proportion of pts maintaining a weighted averagehemoglobin (Hb) ≥9 g/dL without RBC transfusion for ≥12 consecutive months (m; TI12)...Consistent with that, there were 7 cases(7/56; 12.5%) of hepatic veno-occlusive disease; none resulted in end-organ dysfunction, all were relatedto busulfan and all resolved after defibrotide treatment... Exa-cel demonstrated durable clinical benefit for up to 6 y in adults and adolescents withTDT. After exa-cel, iron was successfully removed by IRT with no evidence of iron reaccumulation after IRTcessation. This suggests that in addition to durable TI in 98% of subjects, exa-cel potentially preventstissue iron deposition by restoring iron homeostasis via correction of underlying IE."
Clinical • Beta-Thalassemia • Genetic Disorders • Hepatology • ERFE
November 06, 2024
Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
(ASH 2024)
- "Participants who complete CLIMB-111 were offered enrollment in a 13-yr long term study, CLIMB-131; total follow-up in these 2 studies will be up to 15 yrs after exa-cel infusion. The safety profile of exa-cel remains consistent with myeloablative busulfan conditioning and autologous transplantation. These results confirm the potential for exa-cel to provide a one-time functional cure to patients with TDT."
Clinical • Anemia • Beta-Thalassemia • Dental Disorders • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Hematological Disorders • Neutropenia • Stomatitis • CD34
November 06, 2025
Durable clinical benefits with exagamglogene autotemcel for transfusion-dependent thalassemia and severe sickle cell disease
(DGHO 2025)
- "We report long-term efficacy and safety from the CLIMB THAL-111, CLIMB SCD-121, and CLIMB-131 studies. CLIMB-111 (TDT) and CLIMB-121 (SCD) are ongoing 2-year, Phase 3 studies of exa-cel in pts aged 12-35 y.The CLIMB-111 primary endpoint is transfusion independence defined as the proportion of pts maintaining a weighted average hemoglobin ≥9 g/dL without RBC transfusion for ≥12 consecutive months (mos; TI12). Exa-cel demonstrated durable clinical benefit in TDT and SCD pts and a safety profile consistent with busulfan myeloablative conditioning and autologous transplant. In TDT, there was no evidence of iron reaccumulation after IRT cessation. In SCD, younger age and blood CD34+ cells/µL at BL and pre-apheresis correlated with higher CD34+ cell collection."
Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34
May 16, 2025
DURABLE CLINICAL BENEFITS AND IMPROVEMENT OF TISSUE IRON OVERLOAD WITH EXAGAMGLOGENE AUTOTEMCEL FOR TRANSFUSION-DEPENDENT THALASSEMIA
(EHA 2025)
- "CLIMB-111 pts then enroll in long-term follow-up study CLIMB-131 for up to 15 yrs of follow-up. Exa-cel demonstrated durable clinical benefit in adults and adolescents with TDT and a safety profile consistent with busulfan myeloablative conditioning and autologous transplant. After exa-cel infusion, storage iron can be successfully removed by IRT with no evidence of iron reaccumulation after cessation of IRT. This suggests that in addition to durable transfusion independence, exa-cel has the potential to prevent tissue iron deposition, eliminating the need for chronic IRT, and may preserve end-organ function."
Clinical • Beta-Thalassemia • Genetic Disorders • Hematological Disorders
February 05, 2025
DURABLE CLINICAL BENEFITS IN TRANSFUSION-DEPENDENT Β-THALASSEMIA WITH EXAGAMGLOGENE AUTOTEMCEL
(EBMT 2025)
- P2/3 | "For the 3 pts who did not achieve TI12 in CLIMB-111, 2 achieved TI12 in CLIMB-131 (duration transfusion free: 23.0 and 15.7mos), while 1 stopped transfusion for 4.8mos. Durable transfusion independence was achieved in >94% of pts receiving exa-cel, with associated clinically meaningful and sustained increases in HbF and total Hb for up to 5yrs of available follow-up. Exa-cel's safety profile remains consistent with busulfan myeloablation and autologous transplantation. These findings confirm exa-cel's potential as a one-time functional cure for patients with TDT."
Clinical • Beta-Thalassemia • Genetic Disorders • Neutropenia • Oncology • CD34
December 19, 2024
Durable Clinical Benefits in Transfusion-Dependent Β-Thalassemia with Exagamglogene Autotemcel
(TCT-ASTCT-CIBMTR 2025)
- "For the 3 pts who did not achieve TI12 in CLIMB-111, 2 achieved TI12 in CLIMB-131 (duration transfusion free: 23.0 and 15.7mos), while 1 stopped transfusion for 4.8mos. Durable transfusion independence was achieved in >94% of pts receiving exa-cel, with associated clinically meaningful and sustained increases in HbF and total Hb for up to 5yrs of available follow-up. Exa-cel’s safety profile remains consistent with busulfan myeloablation and autologous transplantation. These findings confirm exa-cel’s potential as a one-time functional cure for patients with TDT ."
Clinical • Beta-Thalassemia • Genetic Disorders • Neutropenia • Oncology • CD34
May 14, 2020
New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001™ for Severe Hemoglobinopathies Accepted for Oral Presentation at the 25th European Hematology Association (EHA) Congress
(GlobeNewswire, CRISPR Therapeutics AG)
- "CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that new data from two ongoing Phase 1/2 clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies have been accepted for an oral presentation at the EHA Congress, which will take place virtually from June 11-14, 2020. An abstract posted online today includes 12 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and 6 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Updated data will be presented at EHA, including longer duration follow-up data for the first two patients treated in these trials and initial data for the second patient treated in the CLIMB-111 trial."
Clinical • Clinical data • Enrollment status • Anemia • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease
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