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Editas Nominates EDIT-401 as Lead In Vivo Therapy for High Cholesterol
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Key Takeaways
Editas named EDIT-401 its lead in vivo candidate, targeting LDL-C reduction in hyperlipidemia.
Preclinical primate studies showed EDIT-401 cut LDL-C by ~90%, outpacing standard therapies.
EDIT plans to initiate early-stage EDIT-401 study by mid-2026, aiming for proof-of-concept data by end-2026.
Editas Medicine (EDIT - Free Report) has nominated EDIT-401 as its lead in vivo development candidate. The experimental, one-time gene editing therapy is designed to significantly reduce LDL cholesterol (LDL-C) levels, marking a key milestone in the company’s efforts to advance in vivo programmable gene editing.
More on EDIT’s New Lead In Vivo Candidate
Editas is developing EDIT-401 to address hyperlipidemia (elevated LDL-C). The therapy leverages the company’s differentiated upregulation approach to directly edit the LDLR gene, boosting protein expression and lowering LDL-C levels. Preclinical studies have shown both strong efficacy and favorable tolerability, underscoring its potential to improve outcomes for patients who remain underserved by current lipid-lowering treatments.
Per Editas, atherosclerotic cardiovascular disease (ASCVD) is the leading cause of death globally and represents a growing financial challenge for the U.S. healthcare system, with costs projected to exceed $300 billion by 2035. Hyperlipidemia is a key driver of ASCVD and affects more than 70 million people in the United States. Despite available therapies, a substantial unmet need persists across multiple high-risk patient groups, underscoring the demand for more effective treatment options.
Shares of Editas have soared 94.5% year to date compared with the industry’s 2.7% growth.
Image Source: Zacks Investment Research
In preclinical non-human primate studies, EDIT-401 achieved about a 90% average reduction in LDL-C, significantly exceeding the 40-60% reduction typically seen with standard-of-care therapies. Designed as a one-time treatment with the possibility of lifelong benefit, the candidate represents a potentially transformative advance in cardiovascular medicine.
Additional Updates From EDIT on Its In Vivo Program
Editas reported that while it continues to optimize candidates within its hematopoietic stem cell program, resources will primarily be directed toward advancing EDIT-401. The company aims to identify and disclose an additional target cell type or tissue by the end of 2025 and plans to submit a regulatory filing seeking permission to begin an early-stage clinical study of EDIT-401 by mid-2026. EDIT expects to generate human proof-of-concept data by year-end 2026.
As of June 30, 2025, the company held $178.5 million in cash, cash equivalents, and marketable securities. It reaffirmed guidance that this cash balance, combined with payments retained under its licensing agreement with Vertex Pharmaceuticals, will fund operations and capital needs into the second quarter of 2027.
In the past 60 days, estimates for CorMedix’s earnings per share have increased from $1.10 to $1.49 for 2025. During the same time, earnings per share estimates for 2026 have increased from $1.46 to $2.16. Year to date, shares of CRMD have surged 80.7%.
CorMedix’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 34.85%.
In the past 60 days, estimates for Pharming Group’s 2025 loss per share have narrowed from 40 cents to 10 cents. For 2026, the estimate for PHAR’s earnings per share has improved from 7 cents to 27 cents. PHAR stock has rallied 39.2% year to date.
Pharming Group’s earnings beat estimates in two of the trailing four reported quarters and missed on the remaining two occasions, delivering an average negative surprise of 39.14%.
In the past 60 days, estimates for Kiniksa Pharmaceuticals’ 2025 earnings per share have increased from 74 cents to $1.03. Earnings per share estimates for 2026 have increased from $1.19 to $1.60 during the same period. KNSA stock has surged 73.3% year to date.
Kiniksa Pharmaceuticals’ earnings beat estimates in two of the trailing four reported quarters and missed on the remaining two occasions, delivering an average negative surprise of 330.56%.
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Editas Nominates EDIT-401 as Lead In Vivo Therapy for High Cholesterol
Key Takeaways
Editas Medicine (EDIT - Free Report) has nominated EDIT-401 as its lead in vivo development candidate. The experimental, one-time gene editing therapy is designed to significantly reduce LDL cholesterol (LDL-C) levels, marking a key milestone in the company’s efforts to advance in vivo programmable gene editing.
More on EDIT’s New Lead In Vivo Candidate
Editas is developing EDIT-401 to address hyperlipidemia (elevated LDL-C). The therapy leverages the company’s differentiated upregulation approach to directly edit the LDLR gene, boosting protein expression and lowering LDL-C levels. Preclinical studies have shown both strong efficacy and favorable tolerability, underscoring its potential to improve outcomes for patients who remain underserved by current lipid-lowering treatments.
Per Editas, atherosclerotic cardiovascular disease (ASCVD) is the leading cause of death globally and represents a growing financial challenge for the U.S. healthcare system, with costs projected to exceed $300 billion by 2035. Hyperlipidemia is a key driver of ASCVD and affects more than 70 million people in the United States. Despite available therapies, a substantial unmet need persists across multiple high-risk patient groups, underscoring the demand for more effective treatment options.
Shares of Editas have soared 94.5% year to date compared with the industry’s 2.7% growth.
Image Source: Zacks Investment Research
In preclinical non-human primate studies, EDIT-401 achieved about a 90% average reduction in LDL-C, significantly exceeding the 40-60% reduction typically seen with standard-of-care therapies. Designed as a one-time treatment with the possibility of lifelong benefit, the candidate represents a potentially transformative advance in cardiovascular medicine.
Additional Updates From EDIT on Its In Vivo Program
Editas reported that while it continues to optimize candidates within its hematopoietic stem cell program, resources will primarily be directed toward advancing EDIT-401. The company aims to identify and disclose an additional target cell type or tissue by the end of 2025 and plans to submit a regulatory filing seeking permission to begin an early-stage clinical study of EDIT-401 by mid-2026. EDIT expects to generate human proof-of-concept data by year-end 2026.
As of June 30, 2025, the company held $178.5 million in cash, cash equivalents, and marketable securities. It reaffirmed guidance that this cash balance, combined with payments retained under its licensing agreement with Vertex Pharmaceuticals, will fund operations and capital needs into the second quarter of 2027.
Editas Medicine, Inc. Price and Consensus
Editas Medicine, Inc. price-consensus-chart | Editas Medicine, Inc. Quote
EDIT’s Zacks Rank and Stocks to Consider
Editas currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are CorMedix (CRMD - Free Report) , Pharming Group (PHAR - Free Report) and Kiniksa Pharmaceuticals (KNSA - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for CorMedix’s earnings per share have increased from $1.10 to $1.49 for 2025. During the same time, earnings per share estimates for 2026 have increased from $1.46 to $2.16. Year to date, shares of CRMD have surged 80.7%.
CorMedix’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 34.85%.
In the past 60 days, estimates for Pharming Group’s 2025 loss per share have narrowed from 40 cents to 10 cents. For 2026, the estimate for PHAR’s earnings per share has improved from 7 cents to 27 cents. PHAR stock has rallied 39.2% year to date.
Pharming Group’s earnings beat estimates in two of the trailing four reported quarters and missed on the remaining two occasions, delivering an average negative surprise of 39.14%.
In the past 60 days, estimates for Kiniksa Pharmaceuticals’ 2025 earnings per share have increased from 74 cents to $1.03. Earnings per share estimates for 2026 have increased from $1.19 to $1.60 during the same period. KNSA stock has surged 73.3% year to date.
Kiniksa Pharmaceuticals’ earnings beat estimates in two of the trailing four reported quarters and missed on the remaining two occasions, delivering an average negative surprise of 330.56%.