Editas Medicine, Inc. · NASDAQ:EDIT
Editas Medicine — 2025 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR
18 slides from Editas Medicine's conference presentation, February 2025
Slide 1: Transplantation and Cellular Therapy Tandem Meetings
Transplantation and Cellular Therapy TANDEM MEETINGS Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR® February 12-15, 2025 Hawai'i Convention Center, Honolulu, HI
Slide 2: Reni-Cel Led to Sustained Hemoglobin Normalization in Severe SCD (RUBY Trial)
Reni-Cel, an Investigational AsCas12a Gene- Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial Mark Walters, MD1, Haydar Frangoul, MD2, Christopher McKinney, MD3, Luis Pineiro, MD4, Mark
Slide 3: Disclosure slide
Disclosure slide Mark Walters, MD • Sanofi – Consultant, received honorarium. This relationship has ended. • Vertex Pharmaceuticals – Advisory committee member, received consulting fee. This is a current financial relationship. • Ensoma – Scientific board member, received consulting fee. This is a c
Slide 4: Sickle cell disease is a life-threatening hematological disorder
Sickle cell disease is a life-threatening hematological disorder SCD is a genetic blood Progressive multi-organ There is an unmet need disorder caused by damage, adverse events, for broadly accessible variants in the HBB and comorbidities transformative treatments gene, which cause reduce quality of
Slide 5: Reni-cel employs AsCas12a editing of HBG1 and HBG2 promoter regions to induce HbF
Reni-cel employs AsCas12a editing of HBG1 and HBG2 promoter regions to induce HbF Renizgamglogene autogedtemcel (reni-cel) is an investigational gene-edited autologous hematopoietic stem cell medicine Utilizing proprietary AsCas12a Targets both HBG1 and HBG2 designed to edit with high efficiency pro
Slide 6: The RUBY trial is designed to evaluate safety, tolerability, and efficacy of reni-cel
The RUBY trial is designed to evaluate the safety, tolerability, and efficacy of reni-cel in patients with severe SCD Design Key inclusion criteria Key exclusion criteria Key endpoints • Phase I/II • 12–50 years • Available 10/10 HLA– • Proportion of patients • International, multicenter matched rel
Slide 7: RUBY trial participants are monitored for 24 months after a single infusion
RUBY trial participants are monitored for 24 months after a single infusion of reni-cel ENGRAFTMENT & MOBILIZATION & DRUG PRODUCT MYELOABLATION & SCREENING 24 MONTH APHERESIS MANUFACTURING RENI-CEL INFUSION FOLLOW-UP Obtain consent HSPC CD34+ cells edited at Myeloablative 24-month follow-up and scre
Slide 8: RUBY patient demographics, baseline characteristics, and treatment characteristics
RUBY patient demographics, baseline characteristics, and treatment characteristics DEMOGRAPHICS AND MOBILIZATION, APHERESIS, BASELINE CHARACTERISTICS (N=28) INFUSION, AND ENGRAFTMENT (N=28) SCD genotype, n (%) Number of mobilization and apheresis cycles,† 2.0 (1.0–4.0) βS/βS 27 (96.4) median (range)
Slide 9: Early, durable correction of anemia with sustained HbF induction after reni-cel infusion
Early, durable correction of anemia with sustained HbF induction were observed after reni-cel infusion 18 17.1 16 14.6 14.9 14.5 14.3 14.1 Mean total hemoglobin (g/dL)* 13.7 13.8 14.0 14 13.4 13.5 Reni-cel 13.0 12.5 infusion 12 11.1 9.8 Markers of hemolysis, 10 Total Hb including absolute HbF 8 reti
Slide 10: Increased percentage of F-cells and MCH-F/F-cell were observed early and were durable
Increased percentage of F-cells and MCH-F/F-cell were observed early and were durable By Month 6 (n=18), mean (SD) percentage of F-cells Mean MCH-F/F-cell increased early and was had increased to 99.3% (0.9%) and levels were maintained above the anti-sickling threshold of maintained at >97% through
Slide 11: 27 of 28 treated patients were VOE-free post-reni-cel infusion
Of 28 treated patients, 27 were VOE-free post-reni-cel infusion as of the data cutoff date Data cutoff date: October 29th, 2024. Left panel ends at informed consent date: 0* is day of informed consent. Right panel starts at infusion date: 0^ is day reni-cel was infused. †A severe VOE requiring medic
Slide 12: PROs: Pain, physical function, and social roles improved after reni-cel infusion
PROs: Mean scores for pain intensity, pain interference, physical function, and social roles and activities were improved after reni-cel infusion A. Pain domains B. Physical and social domains 10 70 70 Mean (SEM) pain interference score Reni-cel Reni-cel Mean (SEM) pain intensity score infusion Pain
Slide 13: Reni-cel safety profile reflected busulfan myeloablative conditioning and autologous HSCT
Reni-cel safety profile reflected busulfan myeloablative conditioning and autologous HSCT TEAE CATEGORY N=28 Number of Number of patients (%) events Any TEAE 28 (100) 618 Any TEAE related to reni-cel* 3 (10.7) 6 Any TEAE related to busulfan 27 (96.4) 322 Any serious TEAE 12 (42.9) 26 Any serious TEA
Slide 14: Conclusions
Conclusions • Patients achieved early correction of anemia / normal Hb, and sustained HbF induction to ≥40% total Hb with pancellular distribution • Markers of hemolysis improved or normalized by Month 6 • 27 of 28 treated patients were VOE-free post-reni-cel infusion as of the data cutoff date •
Slide 15: Acknowledgements
Acknowledgements We would like to acknowledge all patients in the RUBY trial, external principal investigators, and clinical sites. This trial was sponsored by Editas Medicine, Inc., Cambridge, MA, United States. Medical writing and editorial assistance were provided by Porterhouse Medical US and we
Slide 16: Supplemental Information
YASTCT _ @CIBMTR Supplemental Information
Slide 17: Supplement Table 1. Markers of hemolysis
Supplement Table 1. Markers of hemolysis Mean levels of markers of hemolysis, ARC, indirect bilirubin, and lactate dehydrogenase normalized by Month 6 post-reni-cel infusion and were generally maintained or further improved by Month 12 Absolute reticulocyte count Indirect bilirubin Lactate dehydroge
Slide 18: Supplement Figure 1. Allelic editing levels
Supplement Figure 1. Allelic editing levels Patients showed sustained high levels of allelic editing in both peripheral blood nucleated cells and bone marrow–derived CD34+ cells, with mean (SD) editing levels of 75.1% (10.8% [n=8]) and 87.8% (3.35% [n=7]) at Month 12, respectively 100 Bone marrow Me