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Editas Medicine Evercore ISI 8th Annual HealthCONx Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Evercore ISI 8th Annual HealthCONx Conference summary2 Dec, 2025
Strategic focus and program overview
Pivoted to focus on LDLR upregulation using CRISPR, aiming for high-potency, low-cost therapies that could transform cholesterol management.
Achieved a 6x increase in LDL receptor in non-human primates, resulting in a 90% reduction in LDL cholesterol, surpassing effects seen with PCSK9 inhibitors.
Approach leverages natural human variants to identify and de-risk targets for safety and efficacy.
Editing strategy stabilizes LDLR mRNA, increasing protein levels without altering the receptor itself.
Mechanism of action and differentiation
Directly increases LDL receptor levels, breaking through the efficacy ceiling of statins and PCSK9 inhibitors.
One-time infusion could achieve cholesterol levels below 50 mg/dL, regardless of baseline.
Targets broad hypercholesterolemia populations, with initial focus on heterozygous familial hypercholesterolemia (HeFH).
Patient populations and clinical development
HeFH patients (~1 million in the US) are the initial target, with plans to expand to high-risk secondary and primary prevention groups.
Anticipates similar effect sizes across subpopulations, supported by consistent results in various animal models.
Human proof-of-concept data expected by end of 2026, with IND/CTA filing planned for mid-2026.
Human efficacy curve across dosing
FDA concerns on upregulation safety
Cell survival with sustained LDLR upregulation?
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Next Editas Medicine earnings date
Q4 20255 Mar, 2026
Next Editas Medicine earnings date
Q4 20255 Mar, 2026
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Frequently asked questions
Editas Medicine, a genome editing company, focuses on developing transformative genomic medicines to treat a range of serious diseases. The company seeks to address the fundamental flaws in existing approaches to genetic and infectious diseases by developing TALEN and CRISPR genome editing systems that function more efficiently than existing technologies. It has invented lymphocyte-directed gene therapy that uses CRISPR/Cas9 technology to manufacture a novel endonuclease. Editas Medicine, Inc. was incorporated in 2013 and is based in Cambridge, Massachusetts.
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