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Our Commitment to You

We are unwavering in our dedication to engaging patient communities every step of the way, forging partnerships that allow us to deeply understand their perspectives and priorities and develop innovative medicines that truly address their needs. By doing this, we will advance science, improve treatments, and expand access faster than by working alone. Understanding the potential impact of our work, we operate with urgency to translate Entrada’s promising science into life-changing therapies.

The needs, preferences, and insights of the community shape the decisions made in our Board room and drive change throughout every phase of our development. Our leadership team includes individuals with a strong background in patient advocacy and the Duchenne advocacy community’s insights have informed the mutation targets we are currently pursuing.

We are guided by the following belief statements that enable us to translate our commitment into actions that serve those living with serious diseases. Each of us at Entrada will uphold this Commitment through our individual actions so we collectively succeed in making positive differences in patients’ lives.

Patients and care partners are experts who have unique and important insights essential to the process of developing and delivering truly transformational therapies.

We embrace the fact that no one is defined by their medical needs alone. Understanding the experiences of patients and families from a wide range of backgrounds and circumstances will equip us to contribute to supports and solutions that meaningfully improve the quality of patients’ lives around the globe.

We will learn from patients, care partners, and families about the disease that affects their lives, the aspects of it that are most challenging, and the types of and degrees of change that would make the biggest difference to them living as full a life as possible.

We recognize that treatment and care options available to patients and families are constantly being reshaped by science, policies, and economic factors, as well as an individual’s age, health status, preferences, and geographic location. We will stay curious about how all these factors may influence perspectives and priorities over time and in different parts of the world.

By engaging with patients and other community members around the world, our programs are informed by diverse community perspectives. These interactions also challenge and inspire us to give our very best every day.

We begin each new research effort by asking patients and family members about their experiences and build on that knowledge as the program progresses. We’ll use what we learn to inform near-term actions and longer-range plans.

We recognize discrimination and disparities in their many forms add to challenges for people living with serious diseases. To improve the future for all, we work with organizations that foster safe spaces for dialogue, challenge the status quo, and provide mentorship in helping us learn and grow.

We seek to understand patients’ benefit expectations and tolerance for risk and uncertainty to inform the ways we monitor, measure, and assess the ongoing safety and efficacy of therapies we are developing.

We will ask patients and families to identify potential obstacles and/or hardships so our clinical studies aren’t slowed by avoidable problems attracting participants or keeping them involved.

Collaborating with community-based organizations and other stakeholders will advance science, improve treatments, and expand access faster than by working alone.

We honor the trust that patients and families have placed in their advocacy organizations and the expertise and knowledge that resides within their teams. We will work shoulder to shoulder with advocates to speed up solutions, always respecting their independence and autonomy.

We strive to include our learnings from patients and families as core information we provide to health authorities so they too gain a better understanding and can use this information to make decisions about approval, coverage, reimbursement, and other issues that affect patient access.

We participate in collaborations among patient advocacy organizations, academic researchers, clinical experts, and other companies to raise awareness, attract resources, build on what has been learned, and address common pain points that hold back progress for patients.

Genuine relationships are built on a shared sense of purpose, clear and open communications, and treating each person as a valued contributor.

We aim to share timely updates about our plans and research findings. At times, the detailed laws, rules, and policies we must follow may limit how much information we can report. We will be as direct as possible about our limitations and how soon we might be able to share more details.

When we engage with patients, families, and advocates, we’ll describe what we hope to learn and be open to feedback about what we’ve done well and ways we can improve. We will report back on what we heard and how we will apply what we learned.

We strive to communicate about science and drug development using language and imagery that help everyone better understand and relate to our work and form appropriate expectations about it.

The Power of Shared Stories

At Entrada, we believe that your voice and story are essential. Each individual’s unique experiences help us learn more about unmet needs, enabling us to develop medicines that can have the greatest impact. Meet some of the inspiring families who have shared their stories with us.

Connect With Us

Every Entradan is united in our shared passion for connecting with patient communities. We attend and host meetings in order to listen, learn, and work towards solutions. We’d love to connect with you! Contact Sarah Friedhoff, our Head of Patient Advocacy, at patientadvocacy@entradatx.com.

To learn more about how we honor your privacy, click to see our Privacy Notice

  • Franklin learning in lab
  • Franklin with family in lab
  • children in lab coats
  • Max with family at headquarters
  • Max with family in lab
  • Max playing with science equipment

Pictured: Entrada’s annual Family Weekend where families living with Duchenne visit the Entrada’s offices in Boston and meet with the team to share experiences and provide input on our programs.

Community Updates

You are an important part of our community, and we are committed to keeping you informed and connected. Sign up for Community Updates—featuring highlights from our team, news releases, updates on clinical trials and where we have been in the community. We will also share upcoming events we will be part of and other news that may be helpful or of interest to you. Your information will not be shared outside of Entrada, and you may unsubscribe at any time by clicking the link at the bottom of our emails or by reaching out to patientadvocacy@entradatx.com.

Our Commitment to Clinical Studies

We are committed to operating clinical studies that are inclusive and accessible. That’s why we take additional steps to ensure that participants of all backgrounds feel supported throughout the entire process. This includes working with community members and advocacy groups, who have helped to inform our clinical studies and support materials.

Participants’ study-related care will be provided by a study doctor and team with deep experience in the condition being evaluated.

Reasonable study-related expenses, such as travel, accommodation and meals will be covered for study participants in accordance with local regulations. This includes booking assistance for travel and lodging.

We prioritize offering home visits whenever possible during the study to minimize the need for travel and lessen disruption to participants’ lives while assuring safety is not compromised.

We acknowledge that there are disparities in healthcare and in access to clinical studies. We are committed to working with communities to make study participation available to people who have historically been left out of these opportunities.

To learn more, visit our Clinical Studies page.

FAQs About Entrada

  • Entrada is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by developing a new class of genetic medicines that engage intracellular targets that are difficult to access with traditional approaches.
  • We are a dedicated team of experts and leaders in both disease biology and therapeutic development, working with urgency to advance genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases, among others.

  • Our lead programs are in development for the potential treatment of patients living with Duchenne muscular dystrophy who are exon 44, 45, 50 and 51 skipping amenable.
  • In addition to our Duchenne programs, Entrada has partnered with Vertex to develop a clinical-stage program, VX-670, for myotonic dystrophy type 1.
  • We have also generated positive preclinical data, which means studies in animals and in cells, from programs focused on ocular and metabolic diseases. This includes two new programs for the potential treatment of inherited retinal diseases:
    • The first ocular candidate, ENTR-801, is an exon 13 skipping developmental therapy targeting Usher syndrome type 2A (USH2A).
    • We expect to name a second clinical candidate in ocular disease in 2026.

  • Our bodies are made up of cells, so it’s no surprise that 75% of disease-causing targets are located inside them. Because of the vital role cells play, they have excellent defense mechanisms. This helps keep bad things out, but can also keep medicines from getting in. Only about 1-2% of today’s biological medicines are able to reach their intended target inside the cell.
  • Our approach is different. Entrada’s initial therapies in development use phosphorodiamidate morpholino oligomers (PMOs) chemically linked to our proprietary Endosomal Escape Vehicle (EEV™) technology.
  • This technology attaches our proprietary PMO to a unique peptide (our EEV) that acts as a carrier, helping it move past cellular barriers – especially the endosome inside the cell, which is a compartment that usually traps or breaks down foreign materials – and deliver the drug to its intended target inside the cell. Check out this video for a closer look at how our EEV-therapeutics work.

  • Entrada is developing exon skipping therapies for people living with Duchenne muscular dystrophy who are exon 44, 45, 50 and 51 skipping amenable.
  • Our approach is designed to address the underlying cause of Duchenne and help the body make dystrophin protein that is near full-length and functional. Dystrophin is important because it helps keep muscles strong and stable.

  • Our most advanced program is ENTR-601-44 for people living with Duchenne muscular dystrophy who are exon 44 skipping amenable. We first established the potential of ENTR-601-44 through our preclinical work. This means studies in animals and in cells.
  • Based on this early preclinical data, we ran a Phase 1 clinical study of ENTR-601-44 in healthy volunteers, meaning adults assigned male at birth who did not have Duchenne. The goal of this Phase 1 study was to better understand the safety of ENTR-601-44 before beginning studies in people living with Duchenne. We were pleased to see that the Phase 1 study of ENTR-601-44 showed encouraging safety.
  • The preclinical data combined with the Phase 1 results, allowed us to begin studies in people living with Duchenne.
  • We have shared initial data from the first cohort of our Phase 1/2 ELEVATE-44-201 clinical study which showed favorable safety and tolerability, as well as earlier-than-expected functional responses. We will continue to share data and updates on our programs as they become available. View our most recent community newsletter for the latest on our programs and sign up to receive real-time updates.

  • Entrada’s ongoing clinical studies include ELEVATE-44 and ELEVATE-45, which are investigating study drugs for people living with Duchenne muscular dystrophy who are amenable to exon 44 and exon 45 skipping.
  • The studies are currently taking place in the United Kingdom and European Union.
  • To learn more about Entrada’s ELEVATE-44 and ELEVATE-45 clinical studies and potential enrollment opportunities, visit our clinical studies page.
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