CAMBRIDGE, Mass. – Editas Medicine , Inc. (NASDAQ:EDIT), a leader in gene editing technology currently valued at $104 million in market capitalization, has unveiled significant advancements in its in vivo preclinical studies and outlined strategic goals for the next three years. The company announced the achievement of in vivo editing of hematopoietic stem cells (HSCs) in non-human primates, a crucial step towards developing treatments for sickle cell disease and beta thalassemia. Additionally, Editas has successfully edited liver cells in non-human primates and delivered gene editing to two other cell types in humanized mice.
By mid-2025, Editas aims to nominate two in vivo development candidates, one for HSCs targeting sickle cell disease and beta thalasemia, and another for an undisclosed liver condition. The company also plans to present further in vivo HSC data and establish a new in vivo target cell type or tissue by the end of the year.
Strategic priorities through 2027 include submitting at least one investigational new drug (IND) application or clinical trial application (CTA), achieving human in vivo proof of concept in HSC editing for the treatment of sickle cell disease and beta thalassemia, and initiating a late-stage trial for at least one asset.
Editas is maintaining its financial position, with approximately $270 million in cash, cash equivalents, and marketable securities as of December 31, 2024, which is expected to fund operations into the second quarter of 2027. According to InvestingPro data, the company holds more cash than debt on its balance sheet, though it's currently burning through cash at a notable rate. The company's current ratio of 3.75 indicates strong short-term liquidity.
The company’s President and CEO, Dr. Gilmore O’Neill, will present these developments and the company's strategic priorities at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday.
Editas Medicine is at the forefront of developing in vivo gene editing therapies, with a robust pipeline aimed at treating a broad range of diseases. The company holds exclusive licenses for CRISPR/Cas12a and CRISPR/Cas9 genome editing systems for human medicines from the Broad Institute and Harvard University.
This news is based on a press release statement and reflects the company's forward-looking plans. The actual results may vary based on future research outcomes and regulatory processes.
In other recent news, Editas Medicine has undergone significant changes. The company announced the departure of its Chief Medical (TASE:PMCN) Officer and a transition plan. This follows the company's strategic shift towards in vivo gene editing efforts, resulting in the discontinuation of its reni-cel program and a workforce reduction of 65%. Despite these changes, Editas Medicine reported preclinical success in hematopoietic stem cells gene editing, a potential treatment for sickle cell disease and beta thalassemia.
Several analyst firms have adjusted their ratings and price targets in response to these developments. Evercore ISI and Baird maintained an Outperform rating but reduced their price targets for Editas Medicine. Truist Securities and Stifel downgraded the stock from Buy to Hold, citing the termination of the reni-cel program and extended timeline for in vivo therapies. However, BofA Securities and Evercore ISI upgraded the company to Buy and Outperform respectively, citing the company's strategic shift and strong cash position.
These recent developments continue to shape the future direction of Editas Medicine.
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