EDIT-401 preclinical data show ≥90% LDL-C drop in primates (EDIT)
Filing Impact
Filing Sentiment
Form Type
8-K
Rhea-AI Filing Summary
Editas Medicine used this 8-K to share new preclinical results for its in vivo CRISPR candidate EDIT-401, being developed as a potential one-time treatment for hyperlipidemia. In non-human primates, a single dose across 1.5–3.0 mg/kg produced ≥90% mean LDL cholesterol reduction that appeared rapidly and remained durable for about six months.
The company reports this effect was achieved with only 10–40% functional editing of LDLR alleles and at least a six-fold mean increase in hepatic LDLR protein, with no adverse clinical observations at the therapeutically relevant 1.5 mg/kg dose. Mouse pharmacokinetic and pharmacodynamic data suggest dose adjustments may not be needed to achieve LDL-C lowering in heterozygous familial hypercholesterolemia patients, supporting continued advancement toward first-in-human studies.
Positive
- None.
Negative
- None.
Insights
Strong EDIT-401 preclinical LDL-C lowering, but still early-stage data.
Editas Medicine highlights that a single dose of EDIT-401 in non-human primates produced ≥90% mean LDL-C reduction at 1.5–3.0 mg/kg, durable for roughly six months. This was accompanied by a ≥6-fold mean increase in hepatic LDLR protein despite only 10–40% functional editing of LDLR alleles.
The company also notes no adverse clinical observations at the therapeutically relevant 1.5 mg/kg dose, which is encouraging for safety at this stage. Mouse pharmacokinetic and pharmacodynamic data with EDIT-401(mu) suggest dose adjustments may not be required for heterozygous familial hypercholesterolemia, but this remains preclinical and unproven in humans.
Overall, these data support moving EDIT-401 toward first-in-human clinical development, as referenced in the forward-looking statements. Actual impact on the investment case will depend on future regulatory feedback and clinical trial results, which are subject to the uncertainties the company outlines in its risk disclosures.
8-K Event Classification
3 items: 7.01, 8.01, 9.01
3 items
Item 7.01
Regulation FD Disclosure
Disclosure
Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.
Item 8.01
Other Events
Other
Voluntary disclosure of events the company deems important to shareholders but not covered by other items.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, and exhibit attachments filed with this report.
Key Figures
LDL-C reduction in NHPs: ≥90% mean reduction
Functional LDLR editing: 10–40%
Hepatic LDLR protein increase: ≥6-fold mean increase
+3 more
6 metrics
LDL-C reduction in NHPs
≥90% mean reduction
Single EDIT-401 dose across all non-human primate dose groups
Functional LDLR editing
10–40%
Functional editing of LDLR alleles associated with LDL-C lowering
Hepatic LDLR protein increase
≥6-fold mean increase
Observed in non-human primate liver after EDIT-401 dosing
Dose range in NHP study
1.5–3.0 mg/kg
Evaluated dose levels showing durable LDL-C lowering for ~6 months
Therapeutically relevant dose
1.5 mg/kg
No adverse clinical observations reported at this dose in NHPs
LDL-C durability window
≈6 months
Duration of LDL-C lowering in non-human primates after single dose
Key Terms
in vivo CRISPR gene editing, LDL cholesterol (LDL-C), heterozygous familial hypercholesterolemia (HeFH), pharmacokinetics and pharmacodynamics, +2 more
6 terms
in vivo CRISPR gene editing technical
"a Durable In Vivo CRISPR Gene Editing Therapy That Upregulates LDLR Protein"
LDL cholesterol (LDL-C) financial
"a single dose of EDIT-401 achieved 90% or greater mean LDL cholesterol (“LDL-C”) reduction"
heterozygous familial hypercholesterolemia (HeFH) medical
"support that dose adjustments may not be needed to achieve LDL-C lowering in Heterozygous Familial Hypercholesterolemia (HeFH) patients"
pharmacokinetics and pharmacodynamics medical
"data evaluating pharmacokinetics and pharmacodynamics of a single dose of EDIT-401(mu)"
DNA large language prediction models (DNA-LLM) technical
"Data support leveraging DNA large language prediction models (DNA-LLM) to accelerate and streamline the pursuit of gene editing-based strategies"
upregulation strategy technical
"highlight the broader potential and differentiation of our upregulation strategy to generate new medicines"
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
_________________________________________________________________________________________
FORM 8-K
_________________________________________________________________________________________
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): May 14, 2026
_________________________________________________________________________________________
(Exact Name of Registrant as Specified in its Charter)
_________________________________________________________________________________________
| (State or Other Jurisdiction of Incorporation) | (Commission File Number) | (IRS Employer Identification No.) | ||||||
| (Address of Principal Executive Offices) | (Zip Code) | |||||||
Registrant’s telephone number, including area code: (617 ) 401-9000
(Former Name or Former Address, if Changed Since Last Report)
__________________________________________________________________________________________________
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
| Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) | |||||
| Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) | |||||
| Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) | |||||
| Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) | |||||
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||||||
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company o
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. o
Item 7.01 Regulation FD Disclosure.
On May 14, 2026, Editas Medicine, Inc. (the “Company”) issued a press release titled “Editas Medicine Reports New Preclinical Data Demonstrating Progress of EDIT-401 as Potential Treatment for Hyperlipidemia at the American Society of Gene and Cell Therapy 2026 Annual Meeting,” a copy of which is furnished as Exhibit 99.1 hereto.
The information in this Item 7.01, including Exhibit 99.1 attached hereto, is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 8.01 Other Events.
On May 14, 2026, at the 2026 Annual Meeting of the American Society of Gene and Cell Therapy (“ASGCT”), the Company reported new preclinical data supporting the continued advancement of the Company’s lead in vivo development candidate, EDIT-401, and its potential as a one-time treatment for hyperlipidemia, as well as the broader potential of the Company’s differentiated upregulation strategy. The Company reported that a single dose of EDIT-401 achieved 90% or greater mean LDL cholesterol (“LDL-C”) reduction across all dose groups in non-human primates. This reduction was achieved with only moderate levels (10-40%) of functional editing of LDLR alleles and a six or greater-fold mean increase in hepatic LDLR protein. LDL-C lowering was rapid and remained durable across evaluated dose levels (1.5 mg/kg-3.0 mg/kg) through approximately six months. The data demonstrated a promising preclinical safety profile with no adverse clinical observations at the therapeutically relevant dose (1.5 mg/kg). The highest delivery of EDIT-401 was observed in the hepatocytes as compared to other non-target tissues with undetectable oocyte delivery. The Company also reported at ASGCT that data evaluating pharmacokinetics and pharmacodynamics of a single dose of EDIT-401(mu) across multiple dose levels in heterozygous Ldlr loss-of-function mice and wildtype mice support that dose adjustments may not be needed to achieve LDL-C lowering in heterozygous familial hypercholesterolemia (HeFH) patients.
Item 9.01 Financial Statements and Exhibits.
(d)Exhibits
| Exhibit No. | Description | |||||||
| 99.1 | Press release issued by the Company on May 14, 2026* | |||||||
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |||||||
*This exhibit shall be deemed to be furnished and not filed.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| EDITAS MEDICINE, INC. | ||||||||
| Date: May 14, 2026 | By: | /s/ Amy Parison | ||||||
| Amy Parison | ||||||||
| Chief Financial Officer | ||||||||
Exhibit 99.1
Editas Medicine Reports New Preclinical Data Demonstrating Progress of EDIT-401 as Potential Treatment for Hyperlipidemia at the American Society of Gene and Cell Therapy 2026 Annual Meeting
New data demonstrate promising preclinical safety profile and durable LDL-C lowering of ≥90% with single dose of EDIT-401 in non-human primates through ~6 months
CAMBRIDGE, Mass., May 14, 2026 – Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company developing transformative medicines for serious diseases, shared new preclinical data supporting the continued advancement of Editas’ lead in vivo development candidate, EDIT-401, and its potential as a one-time treatment for hyperlipidemia, as well as the broader potential of the Company’s differentiated upregulation strategy. The data is being presented this week at the 2026 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Boston, including one oral presentation and two poster presentations, as well as one oral presentation at TIDES USA 2026: Oligonucleotide and Peptide Therapeutics Conference.
Key EDIT-401 data presented include:
•In an oral presentation at ASGCT, Editas reported that a single dose of EDIT-401 achieved ≥90 percent mean LDL-C reduction across all dose groups in non-human primates (NHPs).
o≥90 percent mean LDL-C reduction was achieved with only moderate levels (10-40 percent) of functional editing of LDLR alleles and ≥6-fold mean increase in hepatic LDLR protein.
oLDL-C lowering was rapid and remained durable across evaluated dose levels (1.5 mg/kg-3.0 mg/kg) through ~6 months.
oPromising preclinical safety profile with no adverse clinical observations at therapeutically relevant dose (1.5 mg/kg).
oThe highest delivery of EDIT-401 was observed in the hepatocytes as compared to other non-target tissues with undetectable oocyte delivery.
•In an oral presentation at TIDES, Editas presented data demonstrating EDIT-401 dose-dependent LDL-C reduction in NHPs.
•In a poster presentation at ASGCT, Editas reported that data evaluating pharmacokinetics and pharmacodynamics of a single dose of EDIT-401(mu) across multiple dose levels in heterozygous Ldlr loss-of-function mice and wildtype mice support that dose adjustments may not be needed to achieve LDL-C lowering in Heterozygous Familial Hypercholesterolemia (HeFH) patients.
Additional in vivo upregulation findings from a poster presentation at ASGCT include:
•Data support leveraging DNA large language prediction models (DNA-LLM) to accelerate and streamline the pursuit of gene editing-based strategies designed to mitigate disease through augmentation of alternate or compensatory pathways and further highlight the broader potential of Editas’ in vivo gene upregulation platform.
“These new EDIT-401 preclinical data, including durability of LDL-C reduction across a range of doses through ~6 months demonstrated in NHPs, strengthen our confidence in EDIT-401 as a potential one-time treatment to deliver meaningful and durable LDL-C lowering and support its continued advancement toward first-in-human clinical development,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “Further, the data presented also highlight the broader potential and differentiation of our upregulation strategy to generate new medicines across multiple disease areas.”
The presentation details are listed below. Abstracts can be accessed on the ASGCT website, and the presentations will be posted on the Editas Medicine website during the conferences.
American Society of Gene and Cell Therapy (ASGCT) 2026 Annual Meeting, May 11-15
Oral Presentation:
Title: Preclinical Development of EDIT-401, a Durable In Vivo CRISPR Gene Editing Therapy That Upregulates LDLR Protein to Lower LDL-C
Session Date and Time: Thursday, May 14, 3:30 p.m. – 5:00 p.m. EDT
Title: Preclinical Development of EDIT-401, a Durable In Vivo CRISPR Gene Editing Therapy That Upregulates LDLR Protein to Lower LDL-C
Session Date and Time: Thursday, May 14, 3:30 p.m. – 5:00 p.m. EDT
Session Title: Gene Therapy for Cardiovascular Diseases
Presentation Room: 206AB
Final Abstract Number: 380
Poster Presentations:
Title: Pharmacokinetics and Pharmacodynamics of In Vivo Gene Editing Therapy for Lowering LDL-C in Mice
Session Date and Time: Thursday, May 14, 5:00 p.m. – 6:30 p.m. EDT
Session Title: Thursday Poster Reception
Presentation Room: Exhibit and Poster Hall
Final Abstract Number: 3423
Title: In Vivo CRISPR-based Disruption of an Important Gene Repressor Element Upregulates a Compensatory Protein to Normalize Disease-Associated Biomarkers in a Knockout Mouse Disease Model
Session Date and Time: Wednesday, May 13, 5:00 p.m. – 6:30 p.m. EDT
Session Title: Wednesday Poster Reception
Presentation Room: Exhibit and Poster Hall
Final Abstract Number: 2324
TIDES USA 2026: Oligonucleotide and Peptide Therapeutics Conference, May 11-14
Oral Presentation:
Title: Transformative LDL Cholesterol Lowering In Vivo CRISPR Gene Editing Approach for Hyperlipidemia and Atherosclerotic Cardiovascular Disease
Session Date and Time: Wednesday, May 13, 8:30 a.m. – 9:00 a.m. EDT
Session Title: mRNA & Genome Editing: Technology & Applications
Title: Transformative LDL Cholesterol Lowering In Vivo CRISPR Gene Editing Approach for Hyperlipidemia and Atherosclerotic Cardiovascular Disease
Session Date and Time: Wednesday, May 13, 8:30 a.m. – 9:00 a.m. EDT
Session Title: mRNA & Genome Editing: Technology & Applications
About Editas Medicine
As a pioneering gene editing company, Editas Medicine is focused on translating the power and potential of CRISPR genome editing systems into a robust pipeline of transformative in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize durable, precision in vivo gene editing medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com.
Forward-Looking Statements
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “target,” “should,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the progress and results of the Company’s preclinical studies and planned clinical trials, including the Company’s expectation to initiate a first-in-human clinical trial of EDIT-401; and the potential of, and expectations for, EDIT-401. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation, timing, progress, and results of preclinical studies and clinical trials; uncertainty regarding availability and timing of results from preclinical studies and clinical trials; uncertainties relating to planned regulatory submissions to initiate clinical trials, including that results of preclinical studies will warrant such submissions or that regulatory agencies may require additional preclinical studies, that regulatory submissions shall occur on the expected timelines and that regulatory authorities will provide clearance for trials to be initiated; and that the Company will not be able to raise funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is
on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent the Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.
###
Investor and Media Contacts:
ir@editasmed.com
media@editasmed.com
FAQ
What did Editas Medicine (EDIT) announce about EDIT-401 in this 8-K?
Editas Medicine reported new preclinical data for EDIT-401, its lead in vivo CRISPR candidate for hyperlipidemia. A single dose produced ≥90% mean LDL-C reduction in non-human primates, with durable effects through about six months and a promising preclinical safety profile at a therapeutically relevant dose.
How effectively did EDIT-401 lower LDL cholesterol in preclinical non-human primate studies?
In non-human primates, a single EDIT-401 dose achieved ≥90% mean LDL-C reduction across all tested dose groups. This LDL-C lowering occurred rapidly and remained durable for roughly six months at doses between 1.5 mg/kg and 3.0 mg/kg, indicating strong preclinical lipid-lowering activity in this model.
What safety findings did Editas Medicine report for EDIT-401 in preclinical studies?
Editas Medicine reported a promising preclinical safety profile for EDIT-401, with no adverse clinical observations at the therapeutically relevant 1.5 mg/kg dose in non-human primates. The highest delivery occurred in hepatocytes, while delivery to non-target tissues was lower and oocyte delivery was undetectable in the reported studies.
How does EDIT-401 achieve LDL-C reduction according to the reported data?
The company states that EDIT-401 achieved ≥90% mean LDL-C reduction with only 10–40% functional editing of LDLR alleles. This was accompanied by a ≥6-fold mean increase in hepatic LDLR protein, consistent with its design as an in vivo CRISPR gene editing therapy that upregulates LDLR to lower LDL cholesterol levels.
What did Editas report about EDIT-401 in models of heterozygous familial hypercholesterolemia (HeFH)?
In mice, Editas presented pharmacokinetic and pharmacodynamic data for a single dose of EDIT-401(mu) across multiple dose levels. These results support that dose adjustments may not be needed to achieve LDL-C lowering in heterozygous familial hypercholesterolemia patients, though this conclusion is based on preclinical murine models, not human data.
How do DNA large language prediction models relate to Editas Medicine’s in vivo upregulation platform?
Editas reported additional in vivo upregulation findings showing that DNA large language prediction models can help accelerate and streamline gene editing strategies. These models are used to design approaches that augment alternate or compensatory pathways, supporting the broader potential of the company’s in vivo gene upregulation platform beyond EDIT-401.