Editas Medicine, Inc. (NASDAQ:EDIT) BofA Securities 2025 Healthcare Conference May 14, 2025 8:15 PM ET Company Participants Amy Parison - CFO Christi Bernett - SVP of IR Conference Call Participants Alec Stranahan - BofA Securities Alec Stranahan Welcome to the last fireside for day 1 of the 2025 Bank of America Healthcare Conference. Thanks for joining this session with Editas. My name is Alec Stranahan. I'm senior biotech analyst at BofA, covering Editas. And I'm pleased to be joined by Amy Parison, Chie ...

Summary of Editas' Conference Call Company Overview - **Company**: Editas Medicine - **Industry**: Biotechnology, specifically focusing on gene editing and therapy Key Points and Arguments Focus on In Vivo Development - Editas aims to be a premier in vivo gene editing company, refining its focus since the CEO's arrival three years ago [5] - The company has generated promising data in in vivo gene editing, particularly for sickle cell disease, which is seen as a significant opportunity [6][7] Market Potential and Treatment Efficacy - In vivo gene editing is expected to expand the market and reduce patient burden compared to ex vivo therapies [6] - The company believes that the efficacy bar for in vivo treatments is on par or better than ex vivo options, allowing for a larger patient population [9] Preclinical Data and Optimization - Editas is applying learnings from its ex vivo program to optimize its in vivo gene editing approach, focusing on delivery mechanisms [10][11] - Proprietary TLNP technology is showing great potential in preclinical settings [11] Business Development and Pipeline Expansion - The company is exploring additional investments and partnerships to expand its pipeline, particularly in in vivo applications [13][17] - Editas is focused on being best-in-class in vivo gene therapy, with a strategic approach to target functional proteins [15][16] Financial Position and Capital Efficiency - Editas has a cash runway extending into Q2 2027, providing breathing room to advance its in vivo programs [19][21] - The company is being capital efficient, particularly in managing wind-down costs from previous programs [26] Licensing Agreements and Intellectual Property - Current licensing agreements remain unaffected by recent legal decisions, and Editas is open to new licensing opportunities [28][29] Upcoming Data and Milestones - Editas presented data at ASGCT, showing over 80% reduction in a disease biomarker in its liver program [32] - The company plans to declare two drug candidates by mid-2025 and file an IND for one candidate in 2026, with potential human proof of mechanism expected by late 2026 to early 2027 [36] Conclusion - Editas is focused on advancing its in vivo gene editing capabilities while maintaining a strong financial position and exploring new partnerships to enhance its pipeline [41]

Company to share in vivo preclinical data demonstrating the successful use of targeted lipid nanoparticles to deliver HBG1/2 promoter editing cargo to hematopoietic stem and progenitor cells (HSPCs) at ASGCT this week Company will also share in vivo preclinical proof of concept to upregulate expression of a target liver protein to meaningfully reduce a common disease-associated biomarker at ASGCT this week and TIDES next week Remains on track to declare two in vivo gene editing development candidates via ge ...

Intellia Therapeutics (NTLA) Q1 2025 Earnings Call May 08, 2025 08:00 AM ET Speaker0 Good morning, and welcome to the Intellia First Quarter twenty twenty five Financial Results Conference Call. My name is Drew, and I will be your conference operator today. Following formal remarks, we will open the call up for a question and answer session. This conference is being recorded at the company's request and will be available on the company's website following the end of the call. As a reminder, all participants ...

Core Insights - Editas Medicine is on track to declare two in vivo editing development candidates by mid-2025, one targeting hematopoietic stem cells (HSCs) and the other targeting liver cells [1][2] - The company has demonstrated preclinical proof of concept in non-human primates and humanized mice, showcasing the potential of its gene upregulation strategy across multiple tissues [3][6] - Editas maintains a strong cash position, with cash, cash equivalents, and marketable securities amounting to $269.9 million as of December 31, 2024, which is expected to fund operations into the second quarter of 2027 [4][17] Business Updates - The company achieved in vivo preclinical proof of concept ahead of schedule, indicating significant progress in its gene editing platform [2] - Editas plans to present further preclinical data for HSCs and liver indications by year-end 2025 [1][6] - The company is also on track to establish an additional target cell type or tissue by the end of 2025 [1][6] Financial Performance - For Q4 2024, Editas reported a net loss of $45.4 million, or $0.55 per share, compared to a net loss of $18.9 million, or $0.23 per share, in Q4 2023 [6][14] - Collaboration and other research and development revenues decreased to $30.6 million in Q4 2024 from $60.0 million in Q4 2023, primarily due to revenue recognized from a license agreement with Vertex Pharmaceuticals [6][14] - For the full year 2024, the net loss attributable to common stockholders was $237.1 million, or $2.88 per share, compared to a net loss of $153.2 million, or $2.02 per share, in 2023 [14][15] Restructuring and Cost Management - The company ended the development of its reni-cel program, leading to a workforce reduction of approximately 65% and restructuring charges of $12.2 million in Q4 2024 [6][7] - Research and development expenses decreased to $48.6 million in Q4 2024 from $69.6 million in Q4 2023, primarily due to reduced costs associated with the reni-cel program [6][14] Upcoming Events - Editas Medicine plans to participate in investor events, including the Leerink Partners Global Biopharma Conference and the Barclays 27th Annual Global Healthcare Conference in March 2024 [9]