Editas Medicine(EDIT)2025-03-05 21:11Preclinical Developments - Achieved in vivo preclinical proof of concept for editing hematopoietic stem cells in non-human primates, a key step towards developing treatments for sickle cell disease and beta thalassemia [4] - Demonstrated effective delivery and meaningful levels of editing in HSCs using proprietary targeted lipid nanoparticles after a single dose in non-human primates [7] - Achieved proof of concept in non-human primates validating high efficiency gene editing in the liver with the first use of AsCas12a delivery by LNP [7] Future Milestones - Anticipated 2025 milestones include declaring two in vivo development candidates, one in HSCs and one in liver, and presenting further in vivo data in both HSCs and liver indications [4] - Expected to establish an additional in vivo target cell type/tissue beyond HSCs and liver by the end of 2025 [8] - Expected to launch clinical trials for multiple in vivo programs, including submitting at least one IND/CTA by mid-2026 [13] Strategic Priorities - Strategic priorities through 2027 include submitting at least one IND/CTA and achieving human in vivo proof of concept in HSC editing for sickle cell disease and beta thalassemia [4] - Company plans to present at the 43rd Annual J.P. Morgan Healthcare Conference on January 15, 2025, discussing new in vivo preclinical data and strategic priorities [11] Financial Position - Company has approximately $270 million in cash, cash equivalents, and marketable securities as of December 31, 2024, with a cash runway extending into Q2 2027 [10] - Company aims to derive additional value from its foundational CRISPR IP through sublicensing and monetization financing [8]