Editas Medicine Achieves In Vivo Gene Editing Milestones, Plans Clinical Push
Key Insights
Editas Medicine achieved in vivo preclinical proof of concept by editing hematopoietic stem cells in non-human primates, a crucial step for sickle cell disease (search) and beta thalassemia (search) treatment.
The company demonstrated in vivo editing of liver cells in non-human primates and delivered to two additional cell types in humanized mice, expanding therapeutic possibilities.
Editas anticipates declaring two in vivo development candidates by mid-2025, one for HSCs and one for the liver, alongside presenting further in vivo data.
Editas Medicine, Inc. (Nasdaq: EDIT) has announced new in vivo preclinical proof-of-concept data, anticipated 2025 key milestones, and three-year strategic priorities. The company achieved in vivo preclinical proof of concept by editing hematopoietic stem cells (HSCs) in non-human primates, a key step toward developing a novel in vivo treatment for sickle cell disease (search) and beta thalassemia (search). Additionally, Editas achieved in vivo editing of liver cells in non-human primates and in vivo delivery to two additional cell types in humanized mice.
Gilmore O'Neill, M.B., M.M.Sc., President and Chief Executive Officer of Editas Medicine, stated, "We are also thrilled to share new in vivo preclinical data highlighting the potential of our gene upregulation strategy across multiple tissues with our 'plug 'n play' program. We believe the ability to provide in vivo gene editing that functions via gene upregulation across tissues holds the potential to significantly expand the addressable therapeutic possibilities for CRISPR-based gene editing and uniquely position Editas as a leader in the field moving forward."
Hematopoietic Stem Cell Editing
Editas Medicine achieved effective delivery and meaningful levels of editing in HSCs with its proprietary targeted lipid nanoparticles (tLNPs) after a single dose of tLNP in non-human primates. The company is currently evaluating further optimized LNP formulations expected to achieve therapeutic editing levels.
Liver Cell Editing
Proof of concept was achieved in non-human primates, validating high-efficiency gene editing in the liver with the first use of AsCas12a (search) delivery by LNP. Furthermore, the company demonstrated its upregulation strategy in mice by increasing a clinically relevant target protein, resulting in a significant disease biomarker reduction for an undisclosed liver target.
Expansion to Other Cells and Tissues
Editas Medicine demonstrated in vivo proof of concept for "plug 'n play" delivery to extrahepatic cell types using the company's proprietary LNP targeting platform at high efficiency in humanized mice, expanding the potential applications of their gene editing technology.
Anticipated Milestones and Strategic Priorities
Editas Medicine anticipates declaring two in vivo development candidates by mid-2025, one in HSCs for the treatment of sickle cell disease (search) and beta thalassemia (search), and one in liver cells for an undisclosed indication. The company also plans to present additional in vivo preclinical editing data in both HSCs and liver cells in large animal models and establish an additional in vivo target cell type/tissue beyond HSCs and the liver by the end of 2025.
Strategic priorities through 2027 include submitting at least one investigational new drug (IND) application/clinical trial application (CTA) by mid-2026, beginning human trials by the second half of 2026, and initiating at least one late-stage clinical trial in the second half of 2027. Editas aims to achieve human in vivo proof of concept in at least one indication by the end of 2026, validating the company's in vivo upregulation strategy in humans.
Financial Position
As of December 31, 2024, Editas Medicine had approximately $270 million in cash, cash equivalents, and marketable securities, with an expected cash runway into the second quarter of 2027.