Editas Medicine (NASDAQ: EDIT) posts potent preclinical LDL-C reductions with EDIT-401

Rhea-AI Filing Summary

Editas Medicine, Inc. filed a report describing new preclinical data for EDIT-401, an experimental one-time gene-editing therapy designed to significantly reduce LDL-cholesterol by increasing LDL receptor levels. At a major gene and cell therapy congress, the company reported that in non-human primates a single dose of EDIT-401 led to LDL-C reductions equal to or exceeding 90% within 48 hours. Similar LDL-C reductions of at least 90% were also seen in mice with high baseline LDL-C and reduced LDL receptor function, with effects maintained over a three-month study. The therapy uses a CRISPR/Cas9 nuclease with dual guide RNAs delivered via lipid nanoparticles to disrupt negative regulatory elements in the 3’ UTR, which Editas says increased LDL receptor protein in primate liver by at least six-fold while requiring only moderate functional editing of LDLR alleles.

Insights

Biotech pipeline and clinical strategy analyst

Preclinical EDIT-401 data show strong LDL-C lowering in animal models but remain early-stage.

Editas Medicine highlights EDIT-401 as a potential one-time treatment for high LDL-cholesterol through upregulation of the LDL receptor. In non-human primates, a single dose produced LDL-C reductions equal to or exceeding 90% within 48 hours, and similar reductions were seen in mouse models with impaired LDL receptor function. These effects were maintained over a three-month mouse study, suggesting durable activity in preclinical settings.

The approach uses CRISPR/Cas9 with dual guide RNAs in a lipid nanoparticle to disrupt negative regulatory elements in the LDLR gene’s 3’ UTR, increasing mRNA stability. The company reports at least a six-fold mean increase in LDL receptor protein in primate liver, achieved with only moderate functional editing of LDLR alleles. While these findings suggest efficient editing and strong target engagement, they are limited to animal studies and do not address human safety or efficacy.

If EDIT-401 progresses, future company updates would need to detail regulatory interactions and any planned clinical development to clarify how these preclinical results may translate to patients. For now, this disclosure mainly enriches understanding of the early-stage cardiometabolic pipeline rather than changing overall financial expectations.

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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): October 9, 2025

_________________________________________________________________________________________

Editas Medicine, Inc.

(Exact Name of Registrant as Specified in its Charter)

_________________________________________________________________________________________

Delaware			001-37687			46-4097528
(State or Other Jurisdiction of Incorporation)			(Commission File Number)			(IRS Employer Identification No.)

11 Hurley Street
Cambridge,			Massachusetts			02141
(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):

o			Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
o			Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
o			Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
o			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
Common Stock, $0.0001 par value per share			EDIT			The Nasdaq Stock Market LLC

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 October 9, 2025, Editas Medicine, Inc. (the “Company”) issued a press release titled “Editas Medicine Reports In Vivo Proof-of-Concept Data for EDIT-401 at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress,” 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 October 9, 2025, at the 32nd Annual European Society of Gene and Cell Therapy Congress, the Company reported in vivo preclinical proof-of-concept data for EDIT-401, an experimental, potential best-in-class, one-time therapy to significantly reduce LDL-cholesterol (“LDL-C”) through upregulation of the LDL receptor (“LDLR”).

In preclinical studies in non-human primates, EDIT-401 achieved LDL-C reductions in non-human primates equal to or exceeding 90% within 48 hours of a single dose. EDIT-401 also achieved LDL-C reductions equal to or exceeding 90% in mice with high baseline LDL-C and reduced LDLR function. LDL-C reductions were maintained in mouse models in a three-month study.

The Company’s EDIT-401 therapeutic strategy includes the use of a CRISPR/Cas9 nuclease and dual guide RNAs with lipid nanoparticle delivery to disrupt negative regulatory elements in the 3’ UTR, increasing mRNA stability enabling potent LDLR upregulation. The Company observed that the LDL-C reductions following EDIT-401 treatment were achieved with an at least six-fold mean increase in LDLR protein in the non-human primate liver, requiring only a moderate level of functional editing of LDLR alleles.

Item 9.01    Financial Statements and Exhibits.

(d)Exhibits

Exhibit No.						Description
99.1						Press release issued by the Company on October 9, 2025*
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: October 9, 2025			By:			/s/ Amy Parison
						Amy Parison
						Chief Financial Officer

FAQ

What did Editas Medicine (EDIT) disclose about EDIT-401 in this report?

Editas Medicine reported in vivo preclinical proof-of-concept data for EDIT-401, describing it as an experimental, potential best-in-class, one-time therapy aimed at significantly reducing LDL-cholesterol by upregulating the LDL receptor.

How much LDL-cholesterol reduction did EDIT-401 achieve in non-human primates?

In preclinical studies in non-human primates, a single dose of EDIT-401 achieved LDL-cholesterol reductions equal to or exceeding 90% within 48 hours.

What were the EDIT-401 results in mouse models reported by Editas Medicine (EDIT)?

In mice with high baseline LDL-cholesterol and reduced LDL receptor function, EDIT-401 produced LDL-C reductions equal to or exceeding 90%, and these reductions were maintained over a three-month study.

What gene-editing strategy does EDIT-401 use according to Editas Medicine (EDIT)?

EDIT-401 uses a CRISPR/Cas9 nuclease and dual guide RNAs delivered by lipid nanoparticles to disrupt negative regulatory elements in the LDL receptor gene’s 3’ UTR, increasing mRNA stability and enabling potent LDL receptor upregulation.

What change in LDL receptor protein did Editas Medicine observe with EDIT-401?

Editas Medicine observed that LDL-cholesterol reductions after EDIT-401 treatment were associated with at least a six-fold mean increase in LDL receptor protein in non-human primate liver, achieved with only a moderate level of functional editing of LDLR alleles.

Is the EDIT-401 data reported by Editas Medicine from human clinical trials?

No. The report describes preclinical in vivo data in non-human primates and mice for EDIT-401; it does not contain human clinical trial results.