Editas Medicine, Inc. · NASDAQ:EDIT

Editas Medicine — ASGCT 29th Annual Meeting 2026

20 slides from Editas Medicine's conference presentation, May 2026

  1. Slide 1: Preclinical development of EDIT-401, a durable in vivo CRISPR gene-editing therapy

    ASGCT 29th Annual Meeting, Boston, MA; May 11–15, 2026 Preclinical development of EDIT-401, a durable in vivo CRISPR gene-editing therapy that upregulates LDLR protein to lower LDL-C Anshul Gupta, DVM, MS Vice President, Nonclinical Development Editas Medicine, Inc., Cambridge, MA, United States. Di

  2. Slide 2: Introduction and Methods

    Introduction and Methods UNDER EMBARGO UNTIL MAY 14, 2026

  3. Slide 3: ASCVD is a serious disease with significant opportunity for a transformative therapy to reduce LDL-C

    ASCVD is a serious disease with significant opportunity for a transformative therapy to reduce LDL-C ASCVD is driven by cholesterol-rich plaque accumulation in the arteries • ASCVD is the primary cause of morbidity and mortality globally1, including in patients with heterozygous familial hypercholes

  4. Slide 4: Therapeutic strategy of LDLR upregulation was informed by human genetics

    Editas’ therapeutic strategy of LDLR upregulation for LDL-C reduction was informed by human genetics Seven Icelandic family members identified as carriers of partial LDLR 3' UTR deletion1 Plasma LDL-C levels1 No adverse impacts on health1 3'UTR deletion carriers LDL-C: • 13–72 mg/dL plasma levels n

  5. Slide 5: EDIT-401 uses a differentiated mechanism of action to reduce LDL-C

    EDIT-401 uses a differentiated mechanism of action to reduce LDL-C EDIT-401 therapeutic strategy for LDLR upregulation • CRISPR/Cas9 nuclease and dual gRNAs disrupt negative regulatory elements of the LDLR gene, increasing mRNA stability, enabling ≥6-fold increase in LDLR protein in NHP1 • This ampl

  6. Slide 6: Studies supporting the preclinical development of EDIT-401

    We conducted a series of studies to support the preclinical development of EDIT-401 • NHP data (presented here)  Pharmacology  Biodistribution  Safety and tolerability • Mouse data (Poster #3423): "Pharmacokinetics and pharmacodynamics of EDIT-401(mu), in vivo gene-editing therapy for lowering LD

  7. Slide 7: Pharmacology

    Pharmacology UNDER EMBARGO UNTIL MAY 14, 2026

  8. Slide 8: CRISPR/Cas9 and dual gRNA strategy with LNP delivery creates a potent approach to LDLR upregulation

    CRISPR/Cas9 and dual gRNA-based strategy with LNP delivery creates a potent approach to LDLR upregulation for LDL-C reduction Functional edits increase LDLR protein • CRISPR/Cas9 nuclease and dual expression and LDL-C uptake gRNAs disrupt negative regulatory elements in the 3' UTR, increasing LDLR p

  9. Slide 9: EDIT-401 achieved >90% mean LDL-C reduction by upregulating LDLR

    EDIT-401 achieved >90% mean LDL-C reduction by upregulating LDLR; Dose Range Finding (DRF) Study data shown EXPEDIT-401, CRISPR/Cas9 nuclease, and dual gRNAs for LDLR upregulation encapsulated in a GalNAc conjugated LNP administered to non-human primates Single dose of EDIT-401 Vehicle 1.5 mg/kg 3 m

  10. Slide 10: EDIT-401 displayed rapid, robust and durable LDL-C reduction

    EDIT-401 displayed rapid, robust and durable LDL-C reduction EDIT-401, CRISPR/Cas9 nuclease, and dual gRNAs for LDLR upregulation encapsulated in a GalNAc conjugated LNP administered to non-human primates Single dose of EDIT-401 Vehicle 1.5 mg/kg 2.0 mg/kg 3.0 mg/kg 40 (n=4) (n=4) (n=4) (n=4) 30 20

  11. Slide 11: EDIT-401 enables LDLR protein increase needed for ≥90% LDL-C reduction in NHPs

    EDIT-401 enables increase in LDLR protein levels needed to achieve ≥90% LDL-C reduction in NHPs (% change from baseline) LDL-C vs LDLR protein 0 R2 = 0.8926  A >6-fold mean increase in -20 LDLR protein in liver resulted in ≥90% LDL-C reduction in NHPs -40 LDL-C -60  Approximately 10%–40% -80 funct

  12. Slide 12: Biodistribution

    Biodistribution UNDER EMBARGO UNTIL MAY 14, 2026

  13. Slide 13: Highest delivery of EDIT-401 observed in liver compared to non-target tissues

    Highest delivery of EDIT-401 observed in liver as compared to non-target tissues (assessed by editing levels) Single dose of EDIT-401 at 1.5 mg/kg, D29 25% Vehicle Non-target tissue editing 1.5 mg/kg • Low editing detected in adrenal 20% gland, spleen and ovary of NHP 15% % Indels • No significant e

  14. Slide 14: High delivery in hepatocytes and undetectable cargo in oocytes at 1.5 mg/kg

    EDIT-401 demonstrates high delivery in hepatocytes and undetectable cargo in oocytes at therapeutically relevant dose of 1.5 mg/kg 24hr ISH 24hr IHC Day 29 IHC Liver (20x) Liver (20x) Cas9 mRNA Nuclei Cas9 Protein Nuclei Ovary (30x) * * oocyte oocyte LDLR Protein Nuclei Cas mRNA Detected by Positivi

  15. Slide 15: Safety and tolerability

    Safety and tolerability UNDER EMBARGO UNTIL MAY 14, 2026

  16. Slide 16: No notable treatment-related liver enzyme elevations or liver histopathology findings

    No notable treatment-related liver enzyme elevations or liver histopathology findings at therapeutically relevant dose of 1.5 mg/kg Group Mean Bilirubin EDIT-401 safety Group Mean ALT 1.0 observations at 600 therapeutically relevant 500 Vehicle (n=9) 0.8 Vehicle (n=9) 1.5 mg/kg (n=14) dose of 1.5 mg

  17. Slide 17: Histopathology shows no changes in EDIT-401 treated NHP livers compared to vehicle

    Histopathology shows no changes in EDIT-401 treated NHP livers compared to vehicle on D29 H&E - Morphology LDLR IHC 40X EDIT-401 data at therapeutically relevant dose of 1.5 mg/kg: Vehicle • No evidence of steatosis or fatty liver was observed, even in areas of high LDLR IHC positivity • Compare to

  18. Slide 18: Conclusions

    Conclusions • EDIT-401 combines Editas' drug development expertise and Genevant’s LNP to deliver a differentiated therapeutic strategy of functional LDLR upregulation • A single dose of EDIT-401 achieved ≥90% mean LDL-C reduction with ≥6-fold mean increase in LDLR protein in the NHP liver, requiring

  19. Slide 19: Next Steps & Timing

    Next Steps & Timing First-in-human Presented preclinical Multi-national study initiated proof-of-concept data Phase I/II from NHP and mouse clinical trial prep Presented in vitro models preclinical data Submit IND/CTA by mid-2026 2025 2026 CTA, Clinical Trial Application; IND, Investigational New Dr

  20. Slide 20: Acknowledgments

    Acknowledgments Many thanks to my coauthors: • Judith Newmark • Paul Wrighton • Parth Amin • Jenny Xie • Meetu Seth • Vikram Soman • Hank Lin • Ameya Apte • Morgan Thompson • Ruhong Dong • Eugenio Marco • Jared Saffie • James Bochicchio • Nassim Ajami • Salu Rizal • Linda Burkly • Wei Zhen We would

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