Editas Medicine, Inc. · NASDAQ:EDIT

Editas Medicine — EHA2024 Hybrid Congress (June 2024)

13 slides from Editas Medicine's conference presentation, Jun 15, 2024

  1. Slide 1: Reni-cel, the first AsCas12a gene-edited cell therapy, in the RUBY trial interim analysis

    Reni-cel, the first AsCas12a gene-edited cell therapy, led to hemoglobin normalization and increased fetal hemoglobin in severe sickle cell disease patients in an interim analysis of the RUBY trial Rabi Hanna1, Haydar Frangoul2, Christopher McKinney3, Luis Pineiro4, Markus Mapara5, Jignesh Dalal6, H

  2. Slide 2: Presenting Author Disclosures

    Presenting Author Disclosures Dr. Rabi Hanna • Sanofi, AbbVie, and Vertex (Consultant); Sobi (Speaker bureau)

  3. Slide 3: Sickle Cell Disease Is a Life-Threatening Hematological Disorder

    Sickle Cell Disease Is a Life-Threatening Hematological Disorder Lifelong complications, multi-organ damage, and comorbidities reduce SCD is a genetic blood disorder caused quality of life and shorten lifespan1–4 by variants in the HBB gene, which cause RBCs to sickle1,2 There remains a continued un

  4. Slide 4: 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 and Correct Anemia Renizgamglogene autogedtemcel (reni-cel) is an investigational gene-edited autologous hematopoietic stem cell medicine Utilizing proprietary AsCas12a Targeting HBG1 and HBG2 promoter to edit with hig

  5. Slide 5: The RUBY Trial is Evaluating the Safety, Tolerability, and Efficacy of Reni-cel

    The RUBY Trial is Evaluating the Safety, Tolerability, and Efficacy of Reni-cel in Patients With Severe SCD Design Key Inclusion Criteria Key Exclusion Criteria Key Endpoints • Phase 1/2/3 • 12–50 years • Available 10/10 HLA– • Proportion of patients • International, matched related donor achieving

  6. Slide 6: Patients Receive a Single Infusion of Reni-cel and are Monitored for 24 Months

    Patients in the RUBY Trial Receive a Single Infusion of Reni-cel and are Monitored for 24 Months MOBILIZATION & DRUG PRODUCT MYELOABLATION & ENGRAFTMENT & SCREENING APHERESIS MANUFACTURING RENI-CEL INFUSION 24 MONTH FOLLOW-UP Obtain consent and HSPC mobilization CD34+ cells edited at HBG1 Myeloablat

  7. Slide 7: All Treated Patients Showed Successful Engraftment

    All Treated Patients Showed Successful Engraftment DEMOGRAPHICS APHERESIS, INFUSION, AND (N=18) (N=18) ENGRAFTMENT Genotype, n (%) Number of mobilization and apheresis cycles†, 2.0 (1.0, 4.0) median (min, max) βS/βS 17 (94.4) Total reni-cel dose administered, × 106 CD34+ 4.6 (2.9, 10.0) βS/β0 1 (5.6

  8. Slide 8: All Patients Treated with Reni-cel are VOE-free

    All Patients Treated with Reni-cel are VOE-free Total No. of Pre-Consent Events VOEs Prior to Informed Consent VOE-free After Reni-cel Infusion Patient 1 9 Patient 2 6 Patient 3 11 Severe VOE† Patient 4 11 Non-severe VOE‡ Patient 5 7 Patient 6 7 Patient 7 8 Patient 8 9 Patients have been Patient 9 4

  9. Slide 9: Rapid and Sustained Improvements in HbF with Normalization of Hb

    Patients had Rapid and Sustained Clinically Meaningful Improvements in HbF (>40%), with Early and Durable Normalization of Hb 17.6 18 16 15.2 14.8 14.7 14.5 Mean Total Hemoglobin (g/dL)* 13.8 14.1 14.3 14.2 HbF 14 Reni-cel 13.5 12.6 13.0 infusion Total Hb 12 11.2 Last RBC 9.6 transfusion, mean† 10 8

  10. Slide 10: Pancellular Distribution of HbF in RBCs Above Anti-Sickling Threshold

    Patients Showed Pancellular Distribution of HbF in RBCs, with Sustained Levels Above the Anti-Sickling Threshold Percentage of F-cells increased early MCH-F/F-cell was sustained above the Reni-cel Reni-cel anti-sickling threshold infusion 20 infusion Mean (SEM) Corpuscular HbF (pg/F-cell) 100 18 16

  11. Slide 11: Safety Profile of Reni-cel Consistent With Myeloablative Conditioning With Busulfan and HSCT

    Safety Profile of Reni-cel Is Consistent With That of Myeloablative Conditioning With Busulfan and HSCT TEAE CATEGORY N=18 Number of Number of patients (%) events Any TEAE 18 (100) 374 Any TEAE related to reni-cel* 1 (5.6) 1 No serious TEAEs were Any TEAE related to busulfan 18 (100) 206 reported as

  12. Slide 12: Conclusions

    Conclusions Reni-cel is an investigational autologous gene-edited medicine that demonstrates promising results for gene editing of the γ-globin gene (HBG1 and HBG2) promoters to induce HbF expression in patients with SCD and is the first clinical use of AsCas12a Robust and clinically meaningful impr

  13. Slide 13: Acknowledgements

    Acknowledgements Thank you to participating patients, their families and caregivers, clinical investigators, and study site teams for their support.

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