IMB-2011010 / Purdue, AbCellera - LARVOL DELTA

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

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

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

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

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

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

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