Editas Medicine : April 2026 Corporate Presentation (16def5)

Published: 2026-04-17 09:32:09 ET EDIT

Published on 04/17/2026 at 09:32 am EDT

April 2026

Differentiated Upregulation Strategy

Novel therapeutic mechanisms unlocked by functionally upregulating protein expression

A leader in transformative

in vivo gene editing

Delivery to Target Tissues

Transformative Therapeutic Potential

Delivery platform enables precise delivery of in vivo gene editing medicines across tissue types (liver and others)

Address significant unmet needs via

one-time, durable treatment with meaningful patient and broader healthcare system impact

EDIT-401: Potential to Transform the Hyperlipidemia Treatment Landscape

A potential best-in-class therapy with unprecedented >90% mean reduction in LDL-C in non-human

primates1, on track to achieve early human proof-of-concept data by end of 2026

LDL-C, low-density lipoprotein cholesterol

1. Editas Medicine. Data on file. 3

EDIT-401 Uniquely Positioned

Intensive, lifelong

reduction of LDL-C (<20mg/dL) provides maximal clinical benefit7

Simplified treatment approach may eliminate multiple therapies with life-long administration8

KOLs confirm >90%

mean LDL-C reduction would be transformative for the management of hyperlipidemia1

Average LDL-C Reduction with Current Treatments vs. EDIT-401 Potential

Time

Baseline

% Reduction

Statins

PCSK9i

EDIT-401

Current standard of care has a mean LDL-C reductions of 40-60%2-6

>90% mean LDL-C reduction in non-human primates1

LDL-C, low-density lipoprotein cholesterol; KOL, key opinion leader

1. Editas Medicine. Data on file. 2. Arca M et al. J Am Heart Assoc. 2023;12 3. Goldenberg et al. Vasc Health Risk Manag. 2009:5 369-376 4. Nawrocki JW et al. Arterioscler Thromb Vasc

Biology 1995;15:678-682. 5. Robinson JG et al. N Engl J Med 2015; 372:1489-1499. 6. Koskinas KC et al. J Am Coll Cardiol 2019;74:2452-62. 7 Gaba et al. Circulation. 2023; 147(16):1192-1203. 4

8. Arnett DK et al. Circulation 2019; 140 (11): e596-e646

Differentiated use of CRISPR nuclease-based technology

Edits

Non-Coding, Regulatory

Regions

to upregulate a wild type allele or functional homolog

Treats diseases by increasing the level of disease mitigating protein

Does not alter sequence of naturally occurring protein

Genotype agnostic

Delivering

Best-in-Class Therapy

Addressing Significant At-Risk Populations

Aligning

Patient Benefits with

System Costs

Robust preclinical efficacy with a >90% mean reduction of LDL-C1

with the potential to achieve very low LDL-C levels

~10 million U.S. patients treated for ASCVD not at goal1 including HeFH and at-risk ASCVD patients

One-time treatment has potential to enable long-term patient

adherence at favorable healthcare system economics

LDL-C, low-density lipoprotein cholesterol; HeFH, heterozygous familial hypercholesterolemia; ASCVD, atherosclerotic cardiovascular disease 6

1. Editas Medicine. Data on file.

Heterozygous Familial Hypercholesteremia

Highly prevalent monogenic genetic disorder that leads to elevated LDL-C2

HeFH

1.2 Million

people are affected by HeFH2

~10-20x

increased risk of ASCVD3,4

97%

of HeFH patients do not meet LDL-C goals6

70 million US patients

with hyperlipidemia1

At Risk ASCVD

Driven by cholesterol-rich plaque accumulation in the arteries

ASCVD

~15 Million

US adults with

established ASCVD1

~33%

patients experience

a second event5

~75%

of patients with ASCVD do not meet LDL-C goals7,8

ASCVD, atherosclerotic cardiovascular disease; HeFH, heterozygous familial hypercholesterolemia; LDL-C, low-density lipoprotein cholesterol;

1. Editas Medicine. Data on file. 2. Akioyamen LE, et al. BMJ Open. 2017;7(9):e016461 3. Khera AV. et al. J Am Coll Cardiol. 2016Jun 7;67(22):2578-89 4. Tada H et al. Eur Heart J. 2017 21;38:1573-

1579 5. Mackinnon et al, CJC 2021 Oct 20;4(2):206-213 6. EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC). Lancet. 2021; 398 (10312): 1713-1725 7. Gu J et al. Am J 7

Prev Cardiol 2022 10: 100336; 8. Klimchak AC et al. Am J Prev Cardiol 2020;1:100010

Limited Benefit

Current Standard of Care is Inadequate and Costly

~75% of ASCVD patients do not achieve LDL-C goals1,2

EDIT-401 is poised to transform the treatment landscape and align clinical economic value

Best in class LDL-C reduction

Single dose

Supports long-term adherence

Decreased healthcare costs

Inconvenient Regimens

Multi-drug approach with chronic, burdensome administration3

Poor Adherence

~40% of patients discontinue

after 6 months4

High Cost

Expenditures expected to reach

>$300 billion by 20355

1. Gu J et al. Am J Prev Cardiol 2022; 10: 100336 2. Klimchak AC et al. Am J Prev Cardiol 2020;1:100010 3. Di Girolamo F, Pellin L, Roni C. Eur J Intern Med. 2025;140 4. Donald DR et al. J Clin Lipidol. 2022;16(3):315-

324 5. Khera R et al. J Am Heart Assoc. 2020;9:e017793; https://doi.org/10.1161/CIR.0000000000001258

EDIT-401 Therapeutic Strategy for LDLR upregulation

Edits non-coding, regulatory regions

~10-40% functional editing achieves >90% mean LDL-C reduction in preclinical studies1

At least 6-fold mean LDLR protein increase in preclinical studies1

Differentiated upregulation strategy allows for potential best-in-class LDL-C reduction

LDL-C, low-density lipoprotein cholesterol; LDLR, low-density lipoprotein receptor 9

1. Editas Medicine. Data on file.

∆ICE carriers

7 Icelandic family members identified as carriers of partial LDLR 3' UTR deletion1

Plasma LDL-C levels1

3' UTR deletion carriers

n = 101,857 Icelandic

non-carriers

Mean plasma LDL-C (mmol/l)

No Adverse Impacts on Health1

LDL-C:

13-72 mg/dL in plasma

LDLR:

⮚

1.5-2.5-fold higher surface LDLR

Safety:

No adverse events

LDL-C, low-density lipoprotein cholesterol; LDLR, low-density lipoprotein receptor; 3' UTR, three prime untranslated region

1. Bjornsson et al. Circ Genom Precis Med 2021;14:e003029 10

Non-human Primates1

Vehicle

1.5 mg/kg

mg/kg

-80

100

LDL-C

(mean % reduction from baseline)

>90% mean LDL-C reduction at each dose level

Single dose

At least 6-fold mean increase in LDLR protein upregulation

No adverse clinical observations noted

Transient increases in liver enzymes resolved within 1 week

Pre-dose

24 h

48 h

D15

D22

D29

Pre-dose LDL-C was averaged across 2 timepoints to account for variability in measurements

LDL-C, low-density lipoprotein cholesterol; LDLR, low-density lipoprotein receptor

1. Editas Medicine. Data on file. 11

>90% mean LDL-C reduction

Single dose

At least 8-fold mean increase in LDLR protein upregulation

Wildtype Mice and Mice Heterozygous for LDLR Loss-of-

Function with High Baseline LDL-C1

500

400

300

WT Vehicle

WT Edited

Ldlr+/- Vehicle

Ldlr+/- Edited

200

100

Weeks Post-Dose

LDL-C mg/dL

Mice on a high-fat diet had ≥3-fold elevated baseline LDL-C

compared with mice on a regular-fat diet. N=5 for all WT and Ldlr+/- groups. Ldlr+/-Edited, 100% mean LDL-C reduction from baseline at 12 weeks; WT Edited, 99% mean LDL-C reduction from baseline at 11 weeks.

LDL-C, low-density lipoprotein cholesterol; LDLR, low-density lipoprotein receptor 12

1. Editas Medicine. Data on file.

Edits Non-Coding Regions

Addresses All Patients**

One-time, Curative Potential

Differentiated Therapeutic Strategy

Functional Upregulation

Genetic Knockdown

Genetic Correction

Non-genetic Knockdown*

Harnessing the power of CRISPR gene editing and differentiated therapeutic strategy to develop transformative medicines for people living with serious diseases

*siRNA, antisense oligos, monoclonal antibody, small molecule

**Mutation-agnostic

EDIT-401: Key Anticipated Milestones

2026

Present additional preclinical data for EDIT-401 by mid-2026

Submit IND / CTA for EDIT-401 by mid-2026

Achieve early in vivo human proof of concept for EDIT-401 by end of 2026

2027

Complete enrollment in clinical trial (dose-finding portion) with topline data results available in 2027

Delivering Best-in-Class Therapy

Addressing Significant At-Risk Populations

Aligning Patient Benefits with System Costs

Programs Positioned for Development

PROGRAM (OR DISEASE CANDIDATE) PRECLINICAL IND/CTA

CARD IO VASCU L AR

ENABLING

EARLY-STAGE

CLINICAL

LATE-STAGE

CLINICAL

DEVELOPMENT

& COMMERCIAL PARTNER

EDIT-401: Hyperlipidemia

HEM OG L OBIN - O PATHIE S

In Vivo HSC Editing - sickle cell disease

In vivo HSC Editing - beta thalassemia

O TH ER O RG AN S AN D TIS SU ES

Other Tissue Upregulation Target

AUT OIM MUNE DIS EASE

αβ T Cells - CD19 HD Allo CAR T

CEL L T H ERAPY

αβ T Cells (14 programs)

γδ T Cells

Disclaimer

Editas Medicine Inc. published this content on April 17, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on April 17, 2026 at 13:31 UTC.