Editas Medicine (EDIT) FY 2025 Conference September 03, 2025 08:00 AM ET Company ParticipantsDana Sheehan - Associate Director - Corporate CommunicationsGilmore O’Neill - Director, President & CEOLinda Burkly - EVP & Chief Scientific OfficerAlec Stranahan - VP - Equity ResearchBill Maughan - MD, Equity Research - BiotechnologyJonathan Miller - MD - Biotech & Pharma Equity ResearchSoumit Roy - MD - HealthcareTony Deng - VP - Biotechnology Equity ResearchConference Call ParticipantsJack Allen - Senior Researc ...

Editas Medicine (EDIT) FY 2025 Conference September 03, 2025 08:00 AM ET Speaker0Our lead development candidate, Edit 401. This webinar is being recorded and can be accessed in the future through this same link or through the Investors section of the company's website. After our prepared remarks, we will open the call to Q&A. To ask a question, please click the raise hand feature in the webinar portal. As a reminder, various remarks that we make during this presentation about the company's future expectatio ...

Editas Medicine (EDIT) FY 2025 Conference September 03, 2025 08:00 AM ET Speaker0Our lead development candidate, Edit 401. This webinar is being recorded and can be accessed in the future through this same link or through the Investors section of the company's website. After our prepared remarks, we will open the call to Q&A. To ask a question, please click the raise hand feature in the webinar portal. As a reminder, various remarks that we make during this presentation about the company's future expectatio ...

Summary of Editas Medicine (EDIT) Webinar on Edit 401 Company Overview - **Company**: Editas Medicine (EDIT) - **Focus**: Development of CRISPR-based gene editing medicines, specifically targeting hyperlipidemia through Edit 401 Key Industry Insights - **Industry**: Gene editing and biopharmaceuticals - **Market Potential**: Atherosclerotic cardiovascular disease (ASCVD) is the leading cause of death globally, with U.S. healthcare expenditures projected to exceed $300 billion by 2035 [5][6] Core Product Insights - **Product**: Edit 401 - **Mechanism**: A one-time, in vivo CRISPR gene editing medicine aimed at significantly reducing LDL cholesterol levels - **Efficacy**: Demonstrated a mean reduction of 90% in LDL cholesterol in preclinical studies, compared to 40%-60% reductions achieved by current standard treatments like statins and PCSK9 inhibitors [4][17] - **Treatment Paradigm**: Potential to transform hyperlipidemia treatment by providing a durable, lifetime reduction in LDL levels, thus reducing cardiovascular event risks [5][17] Clinical Development Plans - **Next Steps**: Progressing Edit 401 towards human proof of concept studies, with expected data by the end of 2026 [5][18] - **Patient Segments**: Initial focus may include patients with heterozygous familial hypercholesterolemia (HeFH) and other refractory segments [23][132] Competitive Advantages - **Unique Approach**: Edit 401 utilizes a proprietary CRISPR-Cas9 enzyme and dual-guide RNAs to target the LDLR 3' untranslated region (UTR), leading to increased LDLR protein expression and enhanced LDL clearance [12][11] - **Safety Profile**: Preclinical studies showed no significant adverse effects, with transient increases in liver enzymes resolving within a week [108][109] Market Dynamics - **Patient Population**: Over 70 million patients in the U.S. suffer from elevated LDL levels, with 75% of patients failing to achieve LDL targets with current therapies [6][42] - **Cost Efficiency**: Edit 401 is expected to reduce overall healthcare costs by minimizing the need for multiple therapies and improving patient compliance [18][17] Additional Considerations - **Long-term Safety**: Ongoing studies will assess the durability of LDL reduction and the potential need for redosing [117][119] - **Regulatory Engagement**: Future patient segment selection for clinical trials will be determined in consultation with regulatory bodies [132] Conclusion - Editas Medicine is positioned to potentially revolutionize the treatment of hyperlipidemia with Edit 401, leveraging advanced gene editing technology to achieve significant LDL reductions and improve patient outcomes while addressing a substantial market need.