Editas Medicine, Inc. · NASDAQ:EDIT
Editas Medicine — EHA 2023 Hybrid Congress
13 slides from Editas Medicine's conference presentation, Jun 10, 2023
Slide 1: EDIT-301 Shows Promising Preliminary Safety and Efficacy Results in the Phase I/II RUBY Trial
EDIT-301 Shows Promising Preliminary Safety and Efficacy Results in the Phase I/II Clinical Trial (RUBY) of Patients With Severe Sickle Cell Disease Using Highly Specific and Efficient AsCas12a Enzyme Rabi Hanna, Haydar Frangoul, Christopher McKinney, Luis Pineiro, Markus Mapara, Kai-Hsin Chang, Mic
Slide 2: Disclosures
Disclosures Dr. Rabi Hanna • Consultant for Sanofi, AbbVie, and Cellularity • Speaker bureau for Sobi 2
Slide 3: SCD Is an Inherited Life-Threatening Hematological Disorder Manifesting Shortly After Birth
SCD Is an Inherited Life-Threatening Hematological Disorder Manifesting Shortly After Birth Lifelong complications, multi-organ damage, and comorbidities impact patient SCD is a genetic blood disorder caused quality of life1,2 by mutations in the HBB gene that cause sickling of RBCs; this leads to I
Slide 4: EDIT-301 Employs AsCas12a to Edit HBG1 and HBG2 Promoters, Leading to HbF Induction
EDIT-301 Employs AsCas12a to Edit HBG1 and HBG2 Promoters, Leading to HbF Induction Utilizing proprietary Targeting AsCas12a HBG1 and HBG2 to edit with high promoters to mimic naturally efficiency and minimize occurring mechanisms of off-targets1 HPFH2 Embryo Fetus Adult HBE HBG2 HBG1 HBD HBB β-glob
Slide 5: Ruby Study of EDIT-301 in Patients With Severe SCD
Ruby Study of EDIT-301 in Patients With Severe SCD Design Key Inclusion Criteria Key Exclusion Criteria Key Endpoints • Phase 1/2 • ~40 patients between • Available genetically- • Proportion of patients 18–50 years of age matched (10/10 HLA) achieving complete • International, multicenter related do
Slide 6: Patient Journey in the Ruby Study
Patient Journey in the Ruby Study MOBILIZATION & DRUG PRODUCT MYELOABLATION & ENGRAFTMENT & 24 SCREENING APHERESIS MANUFACTURING EDIT-301 INFUSION MONTH FOLLOW-UP Obtain consent HSPC CD34+ cells edited at Busulfan 24-month follow-up and screen mobilization and HBG1 and HBG2 myeloablation and for pri
Slide 7: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics DEMOGRAPHICS EDIT-301 (N = 4) Genotype, n bS/bS 4 Sex, n Male 2 Female 2 Age (years), median (min–max) 27.5 (25–31) VOEs pre-study annual rate, median (min–max) 4.8 (3–6) LIC (mg/g of liver), median (min–max) 4.2 (2.5–14.9) Treated patients are homozygous fo
Slide 8: All Treated Patients Successfully Engrafted and Have No VOEs
All Treated Patients Successfully Engrafted and Have No VOEs PATIENT 1 PATIENT 2 PATIENT 3 PATIENT 4 EDIT-301 Total CD34+ (106/kg) 10.0 4.0 4.1 3.7 Neutrophil Engraftment (day)* 23 29 23 24 Platelet Engraftment (day)† 19 37 23 28 Follow-up Duration (months) 10 6 3 2 VOEs Post-EDIT-301 Infusion None
Slide 9: Safety Profile of EDIT-301 Is Consistent With That of HSCT and Myeloablative Conditioning With Busulfan
Safety Profile of EDIT-301 Is Consistent With That of HSCT and Myeloablative Conditioning With Busulfan TEAE CATEGORY EDIT-301 (N = 4) Number of Number of patients events Any TEAE 4 43 • Majority of TEAEs (E = 26) occurred within first 30 days Any TESAE 0 0 after EDIT-301 infusion Any TEAE related t
Slide 10: Patients Show Clinically Meaningful Improvements in HbF Levels With Total Hb Returning to Normal Range
Patients Show Clinically Meaningful Improvements in HbF Levels With Total Hb Returning to the Normal Range in as Early as 4 Months 18 PATIENT 1 16 PATIENT 2 16.4 16.6 Normal EDIT-301 16.0 16.3 16.4 16.4 Normal 16 15.7 range in 14 EDIT-301 range in infusion 15.0 12.7 12.5 males* infusion 12.1 12.4 fe
Slide 11: Pancellular Expression of HbF in RBCs and High Levels of Editing Post-EDIT-301 Infusion
Patients Show Pancellular Expression of HbF in RBCs and High Levels of Editing in Peripheral Blood Nucleated Cells Post-EDIT-301 Infusion HbF Expression is Highly Pancellular High Levels of Editing in Peripheral Blood Nucleated Cells 100 100 EDIT-301 % HBG1 and HBG2 Promoter Editing 90 90 infusion 8
Slide 12: Summary
Summary EDIT-301 is a gene-edited autologous hematopoietic stem cell medicine with a unique genomic modification at the γ-globin gene (HBG1 and HBG2) promoters, mimicking the natural mechanism of HPFH EDIT-301 was well-tolerated by the first 4 patients treated • Safety profile of EDIT-301 is consist
Slide 13: Acknowledgements
Acknowledgements Thank you to participating patients, their families, clinical investigators, and study site teams for their support. 13