Company Description
Entrada Therapeutics, Inc. (Nasdaq: TRDA) is a clinical-stage biopharmaceutical company focused on research and development in biotechnology. The company aims to transform the lives of patients by establishing a new class of medicines that engage intracellular targets that have long been considered inaccessible. Entrada is classified in the professional, scientific, and technical services sector and concentrates on neuromuscular, ocular and other serious diseases where there is significant unmet medical need.
Central to Entrada’s approach is its proprietary Endosomal Escape Vehicle (EEV™) technology. The company describes its EEV-therapeutics as being designed to enable efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues, with the goal of achieving an improved therapeutic index. This modular platform supports both RNA- and protein-based programs and underpins Entrada’s pipeline across neuromuscular and ocular indications, among others.
Neuromuscular disease focus and Duchenne franchise
Entrada is advancing a development portfolio of RNA-based oligonucleotide programs for the potential treatment of people living with Duchenne muscular dystrophy (DMD). According to the company, its lead oligonucleotide programs are in development for patients who are exon 44, 45, 50 and 51 skipping amenable. These product candidates are designed within the EEV framework to engage intracellular targets relevant to DMD.
The company has multiple clinical-stage programs in its Duchenne franchise. ENTR-601-44 is being evaluated in the global Phase 1/2 multiple ascending dose (MAD) ELEVATE-44-201 study in ambulatory patients with DMD who are amenable to exon 44 skipping. Entrada reports that it has completed dosing of Cohort 1 in this study and transitioned to the open-label Phase 2 portion. The company has also described plans for ELEVATE-44-102, a Phase 1b MAD study of ENTR-601-44 in ambulatory and non-ambulatory adults with DMD in the U.S.
ENTR-601-45 is being studied in ELEVATE-45-201, a global Phase 1/2 MAD clinical study in ambulatory patients with DMD who are amenable to exon 45 skipping. Entrada has reported initiation of patient dosing in this trial. For patients amenable to exon 50 skipping, the company has received regulatory authorization in the U.K. to initiate ELEVATE-50-201, a Phase 1/2 MAD clinical study of ENTR-601-50, and has outlined plans to submit regulatory applications in the EU. ENTR-601-51 is another Duchenne program for which the company has stated it expects to submit global regulatory applications.
Ocular and metabolic disease programs
Beyond neuromuscular disease, Entrada is expanding into ocular and metabolic indications. The company reports that it has generated preclinical data from programs focused on ocular and metabolic diseases, including new modalities. It has advanced two ocular programs into lead optimization for inherited retinal diseases and has declared ENTR-801 as its first ocular clinical candidate.
ENTR-801 is described by Entrada as an optimized, proprietary oligonucleotide-based therapy for a subgroup of patients with Usher syndrome type 2A (USH2A) who are amenable to exon 13 skipping. According to the company, ENTR-801 is being designed to restore functional usherin protein production with the goal of preserving photoreceptors and stabilizing retinal architecture and function. Entrada notes that ENTR-801 was selected from a large sequence library based on exon skipping activity, usherin protein production and initial safety data in multiple animal models.
Usher syndrome type 2A is characterized in Entrada’s materials as an inherited eye disease caused by changes in the USH2A gene. In some individuals, mutations in exon 13 prevent production of usherin, a protein essential for photoreceptor health. The company highlights that without usherin, photoreceptors gradually degenerate, leading to progressive vision loss and, in many cases, legal blindness, and that there are currently no approved therapies that address the underlying cause of Usher syndrome. Entrada cites estimates that 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.
Partnerships and external collaborations
Entrada has partnered with Vertex Pharmaceuticals on VX-670, a clinical-stage program for the potential treatment of myotonic dystrophy type 1 (DM1). The company states that VX-670 is being evaluated in a global Phase 1/2 MAD clinical trial in people living with DM1, designed to assess both safety and efficacy. Vertex continues to enroll and dose participants in this study, and Entrada highlights this collaboration as part of its broader strategy to apply its intracellular delivery capabilities to serious genetic diseases.
Platform characteristics and development strategy
Across its communications, Entrada describes its EEV technology as proprietary, versatile and modular. This platform is used to support RNA- and protein-based programs targeting intracellular mechanisms that have historically been difficult to reach. By focusing on intracellular targets and delivery, the company aims to create medicines that can be applied across neuromuscular, ocular and other disease areas.
Entrada emphasizes that its development portfolio includes both clinical-stage and preclinical programs. In neuromuscular disease, the company is progressing multiple exon-skipping oligonucleotide candidates for DMD. In ocular disease, it is advancing inherited retinal disease programs and has indicated plans to nominate additional clinical candidates. The company also notes preclinical work in metabolic disease programs, reflecting a broader application of its platform beyond its initial neuromuscular focus.
Regulatory status and listing
Entrada Therapeutics’ common stock, with a par value of $0.0001 per share, is listed on The Nasdaq Global Market under the trading symbol TRDA, as confirmed in the company’s Form 8-K filings. These filings also identify Entrada as an emerging growth company under applicable U.S. securities regulations.
Community and patient engagement
In addition to its research and development activities, Entrada describes initiatives aimed at supporting the Duchenne community. For example, the company has highlighted its DREAMS Grant Program, which provides funding to non-profit organizations working to advance equity, accessibility and inclusion for individuals living with Duchenne muscular dystrophy. Through such programs, Entrada indicates that its engagement with patients and families extends beyond therapeutic development.
Summary
Overall, Entrada Therapeutics presents itself as a clinical-stage biopharmaceutical company built around a proprietary intracellular delivery platform. Its work centers on RNA- and protein-based therapeutics for neuromuscular conditions such as Duchenne muscular dystrophy, ocular diseases including Usher syndrome type 2A, and other serious disorders. With a pipeline that includes multiple exon-skipping candidates and a partnered program in myotonic dystrophy type 1, Entrada’s stated goal is to develop new medicines for patients whose conditions are driven by intracellular targets that have historically been difficult to address.