Entrada Therapeutics Highlights Progress Across its Portfolio of RNA-based Therapeutics for the Treatment of Neuromuscular and Ocular Diseases

Rhea-AI Summary

Entrada Therapeutics (Nasdaq: TRDA) reported clinical and preclinical progress across its RNA-based neuromuscular and ocular programs and confirmed cash to fund operations into Q3 2027. Key near-term milestones include planned data readouts from the first cohorts of ELEVATE-44-201 in Q2 2026 and ELEVATE-45-201 in mid-2026, initiation of a Phase 1/2 MAD study of ENTR-601-50 by end-2026, and global regulatory submissions for ENTR-601-51 in 2026. Entrada also nominated ENTR-801 as its first ocular clinical candidate for Usher syndrome type 2A and expects a second ocular candidate in 2026. The company will present at the J.P. Morgan Healthcare Conference on Jan 14, 2026.

Positive

• Data readout scheduled: ELEVATE-44-201 Cohort 1 in Q2 2026
• Data readout scheduled: ELEVATE-45-201 Cohort 1 in mid-2026
• ENTR-801 nominated as first ocular clinical candidate for USH2A
• Regulatory progress: FDA granted Rare Pediatric Disease Designation to ENTR-601-44
• Cash runway sufficient into Q3 2027

Negative

• None.

News Market Reaction

-2.14%

1 alert

-2.14% News Effect

On the day this news was published, TRDA declined 2.14%, reflecting a moderate negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

ELEVATE-44-201 Cohort 1 dose: 6 mg/kg ELEVATE-44-201 higher doses: Up to 12 mg/kg and 18 mg/kg ELEVATE-45-201 Cohort 1 dose: 5 mg/kg +5 more

8 metrics

ELEVATE-44-201 Cohort 1 dose 6 mg/kg Global Phase 1/2 MAD study in exon 44–amenable DMD

ELEVATE-44-201 higher doses Up to 12 mg/kg and 18 mg/kg Planned Cohorts 2 and 3 dose levels

ELEVATE-45-201 Cohort 1 dose 5 mg/kg Global Phase 1/2 MAD in exon 45–amenable DMD

ELEVATE-45-201 higher doses Up to 10 mg/kg and 15 mg/kg Planned Cohorts 2 and 3 dose levels

Clinical DMD programs 2026 4 programs ENTR-601-44, -45, -50, -51 expected in clinic

Usher syndrome type 2A population Approximately 15,000 people U.S. and Europe, exon 13–amenable USH2A

Cash runway Into Q3 2027 Company-stated funding horizon

Ocular candidates 2 clinical candidates by 2026 ENTR-801 selected; second candidate planned 2026

Market Reality Check

Price: $10.60 Vol: Volume 166,992 is slightl...

normal vol

$10.60 Last Close

Volume Volume 166,992 is slightly below the 20-day average of 188,915 (relative volume 0.88). normal

Technical Shares at $10.75 are trading above the 200-day MA of $7.57 after the portfolio update.

Peers on Argus

TRDA is up 6.65% while peers show mixed moves: CCCC +8.29%, TNYA +6.68%, CGTX +3...

TRDA is up 6.65% while peers show mixed moves: CCCC +8.29%, TNYA +6.68%, CGTX +3.5%, TLSA +1.35%, and EPRX -2.3%. With no peers in the momentum scanner and no same-day peer headlines, today’s move looks more company-specific to Entrada’s pipeline update.

Historical Context

5 past events · Latest: Dec 17 (Neutral)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 17	Conference presentation	Neutral	-1.3%	Announced upcoming J.P. Morgan Healthcare Conference presentation and webcast details.
Dec 04	Equity inducement grants	Neutral	+1.6%	Granted RSUs and stock options to new hires under Nasdaq inducement rule.
Nov 06	Earnings and pipeline	Neutral	+0.1%	Reported Q3 2025 financials and reiterated DMD program timelines and cash runway.
Oct 28	Investor conferences	Neutral	+0.6%	Outlined participation in late-2025 investor conferences with webcast access.
Sep 05	Community grants	Neutral	+1.3%	Announced DREAMS grants of $50,000 each to two DMD-focused non-profits.

Pattern Detected

Recent news has generally led to modest price reactions, with routine corporate updates and conference announcements prompting small single-day moves.

Recent Company History

Over the last several months, Entrada issued mainly corporate and financial updates, including Q3 2025 results on Nov 6, 2025 highlighting $326.8M in cash and runway into Q3 2027, plus multiple conference and grant announcements. Price reactions to these items were small, typically within a few percent. Today’s broad portfolio progress update, reiterating the cash runway and detailing multiple DMD and ocular milestones for 2026, builds directly on those prior pipeline and financing disclosures.

Regulatory & Risk Context

Active S-3 Shelf · $400,000,000

Shelf Active

Active S-3 Shelf Registration 2025-11-06

$400,000,000 registered capacity

An effective Form S-3 shelf filed on Nov 6, 2025 allows Entrada to offer up to $400,000,000 in securities, including a $150,000,000 ATM program within that total, for general corporate purposes such as R&D and working capital. The shelf had not been used yet, with usage_count 0 reported.

Market Pulse Summary

This announcement outlines a broad clinical plan across Duchenne muscular dystrophy, myotonic dystro...

Analysis

This announcement outlines a broad clinical plan across Duchenne muscular dystrophy, myotonic dystrophy type 1 and inherited retinal diseases, with multiple Phase 1/2 milestones targeted in 2026 and a first ocular candidate, ENTR-801, selected for USH2A. The company reiterated cash runway into Q3 2027, consistent with prior disclosures. Investors following the story may focus on timing and quality of upcoming DMD and ocular data, regulatory designations, and any future capital raises under the existing shelf registration.

Key Terms

phase 1/2, multiple ascending dose (mad), duchenne muscular dystrophy (dmd), myotonic dystrophy type 1 (dm1), +4 more

8 terms

phase 1/2 medical

"global Phase 1/2 multiple ascending dose (MAD) portion of the clinical study"

Phase 1/2 is a combined early-stage clinical trial that first tests a new drug or treatment for safety and the right dose, then quickly expands to check if it shows any signs of working in patients. For investors, results from a Phase 1/2 study offer an early read on both risk and potential reward—like a prototype test that both confirms a product won’t harm users and suggests whether it could sell—helping guide valuation and development decisions.

multiple ascending dose (mad) medical

"global Phase 1/2 multiple ascending dose (MAD) portion of the clinical study"

Multiple ascending dose (MAD) is a research process used to test how a new medicine affects the body when given in increasing amounts over several doses. It helps researchers find the safest and most effective dose before the drug is widely used. For investors, understanding MAD studies is important because successful results can signal progress toward new treatments and potential future profits.

duchenne muscular dystrophy (dmd) medical

"advance multiple clinical programs in people living with Duchenne muscular dystrophy (DMD)"

Duchenne muscular dystrophy (DMD) is a severe, inherited disorder caused by a faulty gene that leaves muscle cells unable to maintain their structure, leading to progressive muscle weakness and loss of mobility, often beginning in childhood. Investors care because DMD is a high unmet-need condition that drives demand for new therapies; successful drugs can command premium pricing and regulatory incentives, while clinical trial failures or safety issues can sharply affect companies and their stock value.

myotonic dystrophy type 1 (dm1) medical

"ongoing clinical progress of its myotonic dystrophy type 1 (DM1) partnership (VX-670)"

A hereditary, progressive disorder that weakens muscles and disrupts other organs such as the heart, lungs and endocrine system; it is caused by a repeat error in a gene that makes the body’s cellular instructions work poorly, like a damaged page in an instruction manual. Investors care because it is a chronic, under-treated disease with clear patient need, meaning successful diagnostics or therapies can change care standards, drive sizable markets and materially affect companies’ valuations.

rare pediatric disease designation regulatory

"In December 2025, the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation"

A rare pediatric disease designation is an official regulatory status given to a drug or therapy that targets a serious or life‑threatening condition primarily affecting children and is uncommon in the population. It matters to investors because the status often brings financial and development perks — such as tax credits, reduced fees, faster review and periods of market protection — which can lower costs, speed approval and improve the commercial outlook; think of it as a VIP pass that makes bringing a scarce, child‑focused treatment to market easier and potentially more profitable.

exon 44 skipping medical

"patients living with DMD who are amenable to exon 44 skipping"

Exon 44 skipping is a genetic therapy approach that tells cells to skip over a specific segment of a gene called exon 44, allowing the cellular machinery to produce a shorter but potentially functional version of a missing or faulty protein. For investors, it matters because success or failure in clinical testing, regulatory approval, and patient uptake can directly affect the commercial value of companies developing this targeted repair method—think of it as a precision patch that can make a broken product usable but carries scientific and regulatory risk.

exon 13 skipping medical

"patients with Usher syndrome type 2A (USH2A), who are amenable to exon 13 skipping"

A targeted genetic approach that causes cells to skip a specific section of a gene called exon 13 when they make a protein, typically by altering how the cell’s messenger RNA is assembled. Like removing a broken chapter from an instruction manual so the rest still reads sensibly, exon 13 skipping can turn a severe genetic defect into a milder one or restore partial protein function. For investors, it matters because it represents a drug development strategy that can change clinical outcomes, trial success chances, regulatory decisions, and commercial value of therapies.

usher syndrome type 2a (ush2a) medical

"for the potential treatment of Usher syndrome type 2A (USH2A)"

A genetic condition that causes moderate-to-severe hearing loss from birth and progressive loss of night and peripheral vision due to degeneration of the retina; the underlying cause is mutations in the USH2A gene. Investors should care because it defines a well‑characterized patient population for diagnostics, therapies and clinical trials, so disease prevalence, regulatory pathways and potential treatment demand directly affect market opportunity and development risk — like knowing which homes in a neighborhood share the same faulty wiring.

AI-generated analysis. Not financial advice.

-- Company on track to report ELEVATE-44-201 data from the first cohort in Q2 2026 and ELEVATE-45-201 data from the first cohort in mid-2026 –

-- Expects to initiate global Phase 1/2 MAD clinical study of ENTR-601-50 by the end of 2026 and to submit global regulatory applications for ENTR-601-51 in 2026 –

-- Expands pipeline with selection of ENTR-801 as first clinical candidate in ocular diseases for the treatment of Usher syndrome type 2A and expects to nominate second clinical candidate in 2026 –

-- Cash runway into Q3 2027 –

-- Entrada to present at the 44^th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026, at 3:45 PM PT (6:45 PM ET) --

BOSTON, Jan. 08, 2026 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA) today reported progress across its robust development portfolio of RNA-based programs for the potential treatment of neuromuscular and ocular diseases.

“In 2025, we strategically positioned Entrada to significantly advance what we believe to be best-in-class therapies for people living with Duchenne muscular dystrophy, and expanded our pipeline into ocular diseases with the selection of our first clinical candidate targeting Usher syndrome, an inherited retinal disorder with a profound unmet clinical need,” said Dipal Doshi, Chief Executive Officer at Entrada Therapeutics. “2026 will be a data-rich year for our Duchenne franchise, with multiple readouts including data from the first cohort of ELEVATE-44-201 expected in the second quarter of 2026 and ELEVATE-45-201 in mid-2026. We also plan to advance our growing development portfolio of RNA-based programs and expect to nominate a second clinical candidate in ocular diseases later this year. With sufficient cash resources available, we believe we are well-positioned to advance and expand our unique pipeline of intracellular therapeutics.”

Entrada highlights the following progress against its goal of building a diverse pipeline of transformative therapeutics that address areas of high unmet need where the Company can have a profound impact for patients and their families:

Clinical-Stage Development Pipeline: Entrada continues to advance multiple clinical programs in people living with Duchenne muscular dystrophy (DMD) in the U.K., EU and U.S. In 2026, the Company expects to have four clinical-stage programs in its DMD franchise (ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51), complementing the ongoing clinical progress of its myotonic dystrophy type 1 (DM1) partnership (VX-670) with Vertex.

• ELEVATE-44-201: The Company completed dosing of Cohort 1 of the global Phase 1/2 multiple ascending dose (MAD) portion of the clinical study of ENTR-601-44 in ambulatory patients living with DMD who are amenable to exon 44 skipping, and transitioned to the open label, Phase 2 portion of the study. The Company is on track to report data from Cohort 1 (6 mg/kg) in the second quarter of 2026, data from Cohort 2 (up to 12 mg/kg) by year-end, and data from Cohort 3 (up to 18 mg/kg) to follow. In December 2025, the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to ENTR-601-44.
• ELEVATE-44-102: The Company expects to initiate a Phase 1b MAD clinical study of ENTR-601-44 in ambulatory and non-ambulatory adults living with DMD in the U.S. in the first half of 2026.
• ELEVATE-45-201: The Company initiated patient dosing in the global Phase 1/2 MAD portion of the clinical study of ENTR-601-45 in ambulatory patients living with DMD who are amenable to exon 45 skipping. The Company is on track to report data from Cohort 1 (5 mg/kg) in mid-2026, with data from Cohort 2 and Cohort 3 (up to 10 mg/kg and 15 mg/kg) to follow.
• ELEVATE-50-201: The Company received regulatory authorization from the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee to initiate a Phase 1/2 MAD clinical study of ENTR-601-50 in ambulatory patients living with DMD who are amenable to exon 50 skipping. The Company expects to submit regulatory applications in the EU for ENTR-601-50 in the second half of 2026 and initiate the study by the end of 2026.
• ENTR-601-51: The Company expects to submit global regulatory applications for ENTR-601-51 in 2026.
• VX-670: Vertex continues to enroll and dose the MAD portion of the global Phase 1/2 clinical trial of VX-670 in people living with DM1, which will assess both safety and efficacy. Vertex is on track to complete enrollment and dosing in the trial in the first half of 2026.
  
  

Expanding Preclinical Pipeline: The Company has generated compelling preclinical data from programs focused on ocular and metabolic diseases, which include new modalities.

The Company has advanced two ocular programs into lead optimization for the potential treatment of inherited retinal diseases. Both programs are novel oligonucleotide-based therapeutics with the potential to address areas of high unmet need. In December 2025, Entrada declared its first ocular clinical candidate, ENTR-801, for the potential treatment of Usher syndrome type 2A (USH2A). The Company plans to announce a second clinical candidate in ocular diseases in 2026.

• ENTR-801: The Company’s first ocular candidate is an optimized, proprietary oligonucleotide-based therapy for the potential treatment of a subgroup of patients with Usher syndrome type 2A (USH2A), who are amenable to exon 13 skipping. The clinical candidate is being designed to restore functional usherin protein production with the goal of preserving photoreceptors (the light-sensing cells in the eye) to stabilize the overall retinal architecture and preserve function. ENTR-801 was selected from a library of 200 sequences based on its robust exon skipping and usherin protein production, as well as initial safety in multiple animal models.
  
  USH2A is an inherited eye disease caused by changes in the USH2A gene. In some people, mutations in exon 13 prevent the body from producing usherin, a protein that is essential for the health of photoreceptors. Without usherin, photoreceptors gradually degenerate, leading to progressive vision loss that often begins in early adulthood and can progress to legal blindness by mid-adulthood. There are currently no approved therapies that address the underlying cause of Usher syndrome. In the United States and Europe, approximately 15,000 people are living with Usher syndrome type 2A who may be amenable to exon 13 skipping.
  
  

Cash Runway: The Company continues to be well-capitalized with cash runway anticipated into Q3 2027.

J.P. Morgan Healthcare Conference Presentation and Webcast
Dipal Doshi will deliver a company presentation at the 44^th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026, at 3:45 PM PT (6:45 PM ET). A live webcast will be available on the Presentations portion of Entrada’s Investor Relations website at https://ir.entradatx.com. The webcast will be archived and available for replay for 30 days after the event.

About Entrada Therapeutics
Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines that engage intracellular targets that have long been considered inaccessible. The Company’s Endosomal Escape Vehicle (EEV™)-therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues, resulting in an improved therapeutic index. Entrada is advancing a robust development portfolio of RNA- and protein-based programs for the potential treatment of neuromuscular, ocular and other diseases, leveraging next-generation EEVs, novel oligonucleotide sequences and an advanced protein engineering platform. The Company’s lead oligonucleotide programs are in development for the potential treatment of people living with Duchenne who are exon 44, 45, 50 and 51 skipping amenable. Entrada has partnered to develop a clinical-stage program, VX-670, for myotonic dystrophy type 1.

For more information about Entrada, please visit our website, www.entradatx.com, and follow us on LinkedIn.

Forward-Looking Statements
This press release contains express and implied forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Entrada’s strategy, future operations, prospects and plans, objectives of management, the validation and differentiation of Entrada’s approach and EEV platform and its ability to provide a potential treatment for patients, expectations regarding Entrada’s Phase 1/2 MAD clinical study of ENTR-601-44, including the timing of data from Cohort 1 in the second quarter of 2026, Cohort 2 by end of 2026 and Cohort 3 to follow, expectations regarding initiation of the planned ELEVATE-44-102 study in the U.S. in the first half of 2026, expectations regarding Entrada’s Phase 1/2 MAD clinical study of ENTR-601-45, including the timing of data from Cohort 1 in mid-2026, with data from Cohort 2 and Cohort 3 to follow, expectations regarding the timing of regulatory filings in the EU for the planned Phase 1/2 MAD clinical study of ENTR-601-50 in the second half of 2026 and initiation by the end of 2026, pending clearance, expectations regarding the timing of global regulatory filings and clearance for the planned clinical study of ENTR-601-51 in 2026, the ability to recruit for and complete global Phase 2 clinical studies of ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51, the potential therapeutic benefits of Entrada’s EEV product candidates and the ability to advance therapeutic candidates in indications beyond neuromuscular disease, including but not limited to ocular disease, expectations regarding the timing of nomination of a second clinical candidate for ocular disease in 2026, the continued development and advancement of ENTR-601-44, ENTR-601-45, ENTR-601-50, and ENTR-601-51 for the potential treatment of DMD and ENTR-801 for the potential treatment of Usher syndrome type 2A and the partnered product candidate VX-670 for the potential treatment of DM1, expectations regarding the progress and success of Entrada’s collaboration with Vertex, including completion of enrollment and dosing of the MAD portion of the global Phase 1/2 study of the VX-670 program in the first half of 2026, the ability to continue to expand and develop additional therapeutic programs and modalities, including further exon skipping programs, and the sufficiency of its cash resources into the third quarter of 2027, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Entrada 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 identification and development of product candidates, including the conduct of research activities and the initiation and completion of preclinical studies and clinical studies; uncertainties as to the availability and timing of results from preclinical and clinical studies; the timing of and Entrada’s ability to submit and obtain regulatory clearance and initiate clinical studies; whether results from preclinical studies or clinical studies will be predictive of the results of later preclinical studies and clinical studies; whether Entrada’s cash resources will be sufficient to fund the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Entrada’s filings with the Securities and Exchange Commission (SEC), including the Company’s most recent Form 10-K and in subsequent filings Entrada may make with the SEC. In addition, the forward-looking statements included in this press release represent Entrada’s views as of the date of this press release. Entrada anticipates that subsequent events and developments will cause its views to change. However, while Entrada may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Entrada’s views as of any date subsequent to the date of this press release.

Investor Contact
Karla MacDonald
Chief Corporate Affairs Officer
kmacdonald@entradatx.com

Patient Advocacy Contact
Sarah Friedhoff
Head of Patient Advocacy
patientadvocacy@entradatx.com

Media Contact
Megan Prock McGrath
CTD Comms, LLC
megan@ctdcomms.com

FAQ

When will Entrada (TRDA) report ELEVATE-44-201 cohort 1 data?

Entrada expects to report ELEVATE-44-201 Cohort 1 (6 mg/kg) data in Q2 2026.

What timeline does Entrada (TRDA) give for ELEVATE-45-201 first cohort data?

The company is on track to report ELEVATE-45-201 Cohort 1 (5 mg/kg) data in mid-2026.

What clinical plans does Entrada (TRDA) have for ENTR-601-50 in 2026?

Entrada received U.K. authorization and expects to submit EU regulatory applications in H2 2026 and to initiate the Phase 1/2 MAD study by end of 2026.

What is ENTR-801 and which patient subgroup does it target for Entrada (TRDA)?

ENTR-801 is Entrada’s first ocular clinical candidate designed for patients with Usher syndrome type 2A amenable to exon 13 skipping.

How long is Entrada’s (TRDA) reported cash runway?

The company reports cash runway anticipated into Q3 2027.