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EDIT Set to Begin First-in-Human Study in Hypercholesterolemia
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Key Takeaways
Editas Medicine received approval to initiate the Strive phase I/II study of EDIT-401 in Australia.
The study will assess the safety and efficacy of a single dose, with patient dosing planned for 2026.
EDIT-401 cut key atherogenic lipoproteins by approximately 90% or more in preclinical primate studies.
Editas Medicine (EDIT - Free Report) announced that it has received approval to initiate the first-in-human phase I/II Strive clinical study of EDIT-401, its investigational in vivo gene-editing candidate for heterozygous familial hypercholesterolemia (HeFH), in Australia.
HeFH is an inherited disorder that causes high LDL cholesterol (LDL-C), or bad cholesterol, from an early age, increasing the risk of heart disease, heart attack and stroke. An estimated 1.2 million people in the United States have HeFH, while more than 70 million have elevated LDL-C, highlighting the significant unmet need for effective treatments.
The initiation of the Strive study marks an important milestone in Editas Medicine’s efforts to advance EDIT-401 into clinical development as a potential treatment for patients who require additional LDL-C reduction.
Year to date, the stock has rallied 38.5% compared with the industry’s 2.7% growth.
Image Source: Zacks Investment Research
Goal of EDIT’s Phase I/II Study on EDIT-401
The Strive study will evaluate the safety, tolerability and efficacy of a single dose of EDIT-401. It will be conducted in two parts.
Part 1 is an open-label, single-ascending-dose study designed to identify suitable dose levels and assess safety. Part 2 will be a randomized, placebo-controlled expansion study to further evaluate the therapy.
The study has received the human research ethics committee’s approval and completed Australia's clinical trial notification process with the Therapeutic Goods Administration. The study has also been submitted for review in New Zealand. Subject to the necessary approvals, patients will be enrolled across both countries, with five clinical trial sites selected.
The company remains on track to begin dosing patients in 2026 and anticipates reporting initial safety and tolerability data in the first quarter of 2027.
EDIT-401 Targets LDL Cholesterol Through Gene Editing
EDIT-401 is an investigational in vivo gene-editing therapy designed to treat hyperlipidemia by directly editing the LDLR gene to increase the production of LDL receptor proteins. These proteins help the body remove LDL cholesterol from the bloodstream.
Unlike conventional cholesterol-lowering treatments that generally require ongoing administration, EDIT-401 is being developed as a single-dose therapy with the potential to deliver long-lasting LDL-C reductions. However, its safety, effectiveness and durability in humans have yet to be established.
In preclinical studies involving non-human primates, EDIT-401 demonstrated average reductions of approximately 90% or greater in several atherogenic lipoproteins, including LDL-C, lipoprotein(a) and apolipoprotein B. These early findings support clinical investigation of the therapy.
EDIT's Zacks Rank & Stocks to Consider
Editas Medicine currently carries a Zacks Rank #3 (Hold).
Over the past 60 days, loss per share estimates for Amarin have narrowed from 65 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 51 cents to 50 cents. AMRN’s shares have lost 14.1% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 62.27%.
Over the past 60 days, earnings per share estimates for Alnylam Pharmaceuticals have decreased from $8.65 to $8.63 for 2026. Over the same period, estimates for 2027 earnings per share have narrowed from $12.13 to $12.04. ALNY’s shares have plunged 43.5% year to date.
Alnylam Pharmaceuticals’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 27.58%.
Over the past 60 days, loss per share estimates for Aldeyra Therapeutics have narrowed from 43 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 22 cents to 16 cents. ALDX’s shares have plunged 81.7% year to date.
Aldeyra Therapeutics’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 29.25%.
View the Zacks #1 Rank List
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EDIT Set to Begin First-in-Human Study in Hypercholesterolemia
Key Takeaways
Editas Medicine (EDIT - Free Report) announced that it has received approval to initiate the first-in-human phase I/II Strive clinical study of EDIT-401, its investigational in vivo gene-editing candidate for heterozygous familial hypercholesterolemia (HeFH), in Australia.
HeFH is an inherited disorder that causes high LDL cholesterol (LDL-C), or bad cholesterol, from an early age, increasing the risk of heart disease, heart attack and stroke. An estimated 1.2 million people in the United States have HeFH, while more than 70 million have elevated LDL-C, highlighting the significant unmet need for effective treatments.
The initiation of the Strive study marks an important milestone in Editas Medicine’s efforts to advance EDIT-401 into clinical development as a potential treatment for patients who require additional LDL-C reduction.
Year to date, the stock has rallied 38.5% compared with the industry’s 2.7% growth.
Image Source: Zacks Investment Research
Goal of EDIT’s Phase I/II Study on EDIT-401
The Strive study will evaluate the safety, tolerability and efficacy of a single dose of EDIT-401. It will be conducted in two parts.
Part 1 is an open-label, single-ascending-dose study designed to identify suitable dose levels and assess safety. Part 2 will be a randomized, placebo-controlled expansion study to further evaluate the therapy.
The study has received the human research ethics committee’s approval and completed Australia's clinical trial notification process with the Therapeutic Goods Administration. The study has also been submitted for review in New Zealand. Subject to the necessary approvals, patients will be enrolled across both countries, with five clinical trial sites selected.
The company remains on track to begin dosing patients in 2026 and anticipates reporting initial safety and tolerability data in the first quarter of 2027.
EDIT-401 Targets LDL Cholesterol Through Gene Editing
EDIT-401 is an investigational in vivo gene-editing therapy designed to treat hyperlipidemia by directly editing the LDLR gene to increase the production of LDL receptor proteins. These proteins help the body remove LDL cholesterol from the bloodstream.
Unlike conventional cholesterol-lowering treatments that generally require ongoing administration, EDIT-401 is being developed as a single-dose therapy with the potential to deliver long-lasting LDL-C reductions. However, its safety, effectiveness and durability in humans have yet to be established.
In preclinical studies involving non-human primates, EDIT-401 demonstrated average reductions of approximately 90% or greater in several atherogenic lipoproteins, including LDL-C, lipoprotein(a) and apolipoprotein B. These early findings support clinical investigation of the therapy.
EDIT's Zacks Rank & Stocks to Consider
Editas Medicine currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Amarin (AMRN - Free Report) and Alnylam Pharmaceuticals (ALNY - Free Report) , currently sporting a Zacks Rank #1 (Strong Buy) each, and Aldeyra Therapeutics (ALDX - Free Report) , carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, loss per share estimates for Amarin have narrowed from 65 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 51 cents to 50 cents. AMRN’s shares have lost 14.1% year to date.
Amarin’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 62.27%.
Over the past 60 days, earnings per share estimates for Alnylam Pharmaceuticals have decreased from $8.65 to $8.63 for 2026. Over the same period, estimates for 2027 earnings per share have narrowed from $12.13 to $12.04. ALNY’s shares have plunged 43.5% year to date.
Alnylam Pharmaceuticals’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 27.58%.
Over the past 60 days, loss per share estimates for Aldeyra Therapeutics have narrowed from 43 cents to 39 cents for 2026. Over the same period, estimates for 2027 loss per share have narrowed from 22 cents to 16 cents. ALDX’s shares have plunged 81.7% year to date.
Aldeyra Therapeutics’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 29.25%.