Editas Medicine, Inc. · NASDAQ:EDIT
Editas Medicine — Investor Presentation (February 2025)
20 slides from Editas Medicine's investor presentation, February 2025
Slide 1: Pioneering the Possible in Gene Editing
Stephanie and Tristan Pioneering the Possible in Gene Editing February 2025 1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘pote
Slide 3: In vivo gene editing will transform human therapeutics
In vivo gene editing – a simple IV infusion to cure a disease – will transform human therapeutics globally to the same degree that mobile smart phones have transformed the communications business around the world. 3
Slide 4: There is a Solution to the Recent Challenges of Gene Therapy and Gene Editing
There is a Solution to the Recent Challenges of Gene Therapy and Gene Editing Problem Solution • Recent slow launches • Differentiated therapeutic approach to diseases • Low total addressable markets (TAMs) • Diseases with higher TAMs • Reimbursement challenges • Simple, scalable, lower cost of • Hi
Slide 5: Has the solution
Has the solution A different approach focused on functional upregulation treatment strategy (proven by reni-cel). Solved for delivery using proprietary, targeted LNPs (tLNPs) that allow targeting of multiple tissues, including HSCs, liver, and other tissues using “plug ‘n play” process. Defined path
Slide 6: Editas' Differentiated In Vivo Gene Editing Upregulation Strategy
Editas’ Differentiated In Vivo Gene Editing Upregulation Strategy Designed to Deliver First-to-Market and Best-in-Class Curative Medicines for Genetic Diseases Other Approaches Therapeutic Knockdown Gene correction Functional upregulation* strategy or Gene Editing approach siRNA, antisense oligos, N
Slide 7: Reni-cel Provides Proof of Concept for Functional Upregulation Strategy
Reni-cel Provides Proof of Concept for Functional Upregulation Strategy and Validates Editing the HBG 1/2 Promoter • Reni-cel treatment showed promising results, with robust and clinically meaningful improvements, for gene editing at the HBG1/2 promoters with AsCas12a • Patients achieved early corre
Slide 8: Preclinical POC Data in In Vivo HSC Editing Leverages Editas' Expertise
Preclinical POC Data in In Vivo HSC Editing Leverages Editas’ Gene Editing Expertise and Provides Foundation for LNP Platform Potential for Best-in Class, First-in-Class In Vivo Medicine for Sickle Cell Disease and Beta Thalassemia Proprietary LNP Platform Leveraging reni-cel experience with validat
Slide 9: High Levels of In Vivo HBG1/2 Gene Editing Achieved in HSPCs
High Levels of In Vivo HBG1/2 Gene Editing Achieved in HSPCs with Optimization of a Proprietary LNP and Novel Targeting Strategy in Mice with Human HSPC LNP optimization LNP3 (moiety 2) HbF induction in HBG1/2 editing in HSPC HBG1/2 editing in HSCs erythroid cells 50 40 40 40 ~29% 50 ~40% % HBG1/2 i
Slide 10: High Efficiency HSC Delivery Approaching Therapeutic Editing Levels in NHPs
High Efficiency HSC Delivery Approaching Therapeutic Editing Levels Achieved After a Single Dose of LNP2 in Non-human Primates GFP mRNA delivery and HBG1/2 editing of HSCs after single dose of LNP2 (moiety 2) ~75% GFP+ HSCs observed 24 hours after ~17% HBG1/2 editing in HSCs observed 7 days a single
Slide 11: Preclinical NHP PoC Validates High Efficiency Genomic Editing in Liver
Preclinical NHP PoC Validates High Efficiency Genomic Editing in Liver with First Use of AsCas12a Nuclease Delivery by Lipid Nanoparticle Maximum editing in liver with negligible Robust serum biomarker reduction from editing in non-target tissues (PoC Tool) the baseline (~80%) Editing data generated
Slide 12: In Vivo Preclinical Proof of Upregulation Strategy Confirmed
In Vivo Preclinical Proof of Upregulation Strategy Confirmed by Clinically Relevant Target Protein Increase Resulting in Significant Disease Biomarker Reduction in Undisclosed Target 1 or Target Protein Disease Biomarker Upregulation Reduction 5 0 Fold Change Target Protein Change from Baseline (%)
Slide 13: In vivo PoC for Plug 'n Play Delivery to Extrahepatic Cell Types
In vivo PoC for Plug ‘n Play Delivery to Extrahepatic Cell Types Achieved with Editas’ Proprietary LNP Targeting Platform Specific Delivery of GFP to Target Cells in Humanized Mice Cell type 1 Cell type 2 Cell type 3 In vivo targeting to three extrahepatic cell types at ≥80% efficiency with our plug
Slide 14: A Capital Efficient In Vivo Gene Editing Company
A Capital Efficient In Vivo Gene Editing Company Continued Execution of our Focus to Leverage our Foundational IP Estate for Access to Non-Dilutive Capital Provides upfront cash of $57M via October 03, 2024 non-dilutive financing Editas Medicine Announces $50+ Million Monetization Financing with DRI
Slide 15: Editas' Partnerships Validate Science and Value of IP Estate
Editas’ Partnerships Validate Science and Value of IP Estate Editas is well positioned to capture value of IP and leverage non-dilutive financing to focus resources on in vivo pipeline development Current Collaborators Future IP Licensing and Partners Opportunities Editas’ Partners are Poised to Ach
Slide 16: 2025 Key Anticipated Milestones
2025 Key Anticipated Milestones Declare Two in vivo Development Candidates by Mid-2025 • Candidate in hematopoietic stem cells (HSCs) for the treatment of beta thalassemia and sickle cell disease • Candidate in liver cells for an undisclosed indication Establish One Additional Target Tissue • Disclo
Slide 17: 2025-2027 Strategic Priorities
2025-2027 Strategic Priorities Launch Clinical Trials for Multiple in vivo Programs in Multiple Tissues • Submit at least one IND/CTAs by mid-2026 • Begin at least one human clinical trial by 2H 2026 • Potential for at least one late-stage clinical trial in 2H 2027 Achieve in vivo Human Proof of Con
Slide 18: A different approach focused on functional upregulation treatment strategy
A different approach focused on functional upregulation treatment strategy (proven by reni-cel). Solved for delivery using proprietary, targeted LNPs (tLNPs) that allow targeting of multiple tissues, including HSCs, liver, and other tissues using “plug ‘n play” process. Defined path to rapid develop
Slide 19: Additional Information
Additional Information
Slide 20: Programs Positioned for Development
Programs Positioned for Development PROGRAM (OR DISEASE CANDIDATE) PRECLINICAL IND ENABLING EARLY-STAGE LATE-STAGE DEVELOPMENT CLINICAL CLINICAL & COMMERCIAL PARTNER HEM O G LO BIN- O P AT HI ES In Vivo HSC Editing – sickle cell disease In vivo HSC Editing – beta thalassemia O T HER O RG ANS & T ISS
Editas Medicine — Investor Presentation (February 2025) on 6ix