Editas Shifts Focus to In Vivo Gene Editing CRISPR Bi Investing.com

CAMBRIDGE, MASS. – Publisher Medicine Inc. (NASDAQ: ), a gene-editing company currently valued at about $157 million, announced a strategic shift to prioritize its in vivo CRISPR gene-editing programs, following recent preclinical successes. The company is poised to extend its cash runway in the second quarter of 2027 and plans to achieve human proof of concept for its in vivo treatments within about two years. According to InvestingPro According to the data, the company maintains a strong liquidity position with more cash than debt on its balance sheet, although analysts note that it is quickly burning through available funds.

Editas reported significant advances in in vivo gene editing, including a preclinical proof of concept for editing the HBG1/2 promoter in hematopoietic stem cells (HSCs) in a humanized mouse model, a potential treatment for sickle cell disease and beta thalassemia. This was achieved with a single dose of a targeted lipid nanoparticle (tLNP) formulation. Additionally, the company has demonstrated highly efficient liver editing in non-human primates.

As part of this strategic realignment, Editas is suspending the development of its reni-cel program due to the inability to secure a commercial partner. The company will consult with clinical trial sites, regulators and other stakeholders to determine future steps for patients enrolled in the RUBI and EditHAL trials. InvestingPro analysis reveals that the company’s financial health rating is currently rated as ‘WEAK’, with particular concern regarding profitability metrics. Subscribers can access 14 additional tips and comprehensive financial analysis through the Research Report.

To support the transition, Editas is launching cost-saving measures, including reducing its workforce by approximately 65% ​​over the next six months. This will include changes to the management team, with several members including the boss Medically (TASE:) Officer Baisong Mei, MD, Ph.D., is leaving the company. Emma Reeve and Meeta Chatterjee, Ph.D., will resign from the Board of Directors, and Jessica Hopfield, Ph.D., will serve as Chair of the Board.

Editas President and CEO, Gilmore O’Neill, MB, MMSc., thanked patients, investigators and staff for their dedication to the development of drugs like reni-cel and emphasized the company’s commitment to advancing in vivo treatments for sickle cell disease and beta thalassemia.

Further preclinical data and development timelines for these in vivo gene editing programs are expected to be shared in the first quarter of 2025. The company believes its in vivo gene editing capabilities could significantly expand therapeutic options and position Editas as a leader in the field. With the stock currently trading near its 52-week low of $1.89 and InvestingProFair value analysis suggesting a stock is undervalued Investors looking for in-depth insights can access comprehensive valuation metrics and expert analysis through InvestingPro’s in-depth research reports, available on over 1,400 US stocks.

The information in this article is based on a press release from Editas Medicine.

In other recent news, Editas Medicine was the focus of several analyst adjustments after presenting updated data on its Reni-cel program. Wells Fargo (NISE:) downgraded the company to Equal Weight, citing competing data from another company that could potentially affect Reni-cel’s market position. Even so, Wells Fargo noted that Editas Medicine’s in vivo sickle cell disease program remains a strong contender in its field.

BofA Securities and Evercore ISI upgraded Editas Medicine to Buy and Outperform, with Evercore ISI setting a $7.00 price target. The upgrades reflect the companies’ positive outlook on the company’s strategic shift to focus on its in vivo hematopoietic stem cell program and its strong cash position. Chardan Capital Markets maintained its buy rating and $12.00 price target, reaffirming its positive view on the company.

These recent developments follow the company’s announcement of promising results from RUBI’s ongoing clinical trial for the treatment of severe sickle cell disease, Reni-cel. The trial showed significant improvements in patient health, with rapid and sustained increases in fetal hemoglobin levels. However, the company plans to license its Reni-cel therapy to conserve cash and allocate resources more efficiently.

Editas Medicine also reported third quarter operating expenses of $65.7 million and a cash balance of $265.1 million, supported by a $57 million cash advance from the sale of certain future license fees and payments under the Cas9 license agreement with Vertek Pharmaceuticals (NASDAQ:) . The company anticipates that its current cash reserves will fund operations in the second quarter of 2026.

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