EUCOPE Member Spotlight: Q&A with Sarah Boyce, President and Chief Executive Officer at Avidity Biosciences

Every month, EUCOPE spotlights a member company and the great work they’re doing to advance the life sciences industry and drive innovation to serve patients better. In February, we spoke with Sarah Boyce, President and Chief Executive Officer at Avidity Biosciences.

Tell us about your organisation and its mission and how you drive innovation internally?

Avidity’s mission is to revolutionize the delivery of RNA therapeutics to make a profound difference in the lives of people living with serious rare diseases, many of whom have no or limited treatment options. Utilizing our proprietary Antibody Oligonucleotide Conjugates (AOCs™) technology platform, Avidity is proud to be the first company to demonstrate the successful targeted delivery of RNA to muscle in clinical trials. This groundbreaking accomplishment in the RNA field has led to positive and consistent data across all three of our clinical development programs for three different types of rare muscle diseases: Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). These robust data are promising and a significant step forward for our company, but more importantly the DMD44, DM1 and FSHD communities, which currently face many daily challenges and suffer from significant unmet medical needs.

We also believe in the broad and disruptive potential of the AOC platform, beyond rare neuromuscular diseases and into areas such as precision cardiology and immunology. We continue to expand the possibilities of our AOC platform through internal discovery efforts, and in 2024 announced the advancement of our first two wholly owned development candidates targeting rare genetic cardiomyopathies and made important progress in the development of next-generation technology. As industry leaders in RNA research, we continue to make innovations to our AOC technology to expand the possibilities of RNA delivery. These advancements further our mission to bring much needed therapies to people living with serious rare diseases as quickly as possible.

How do your organisation’s activities help patients now and into the future?

We are transforming RNA delivery and the rare disease space by directly targeting the underlying cause of serious diseases. This means lots of continued hard work ahead, but our progress thus far is exciting for us and the global patient communities we serve. We saw clearly how our potential to help patients expanded when we became the first company to successfully deliver RNA to muscle cells in a targeted fashion leveraging our AOC platform. We have repeated this success across all three of our clinical stage rare neuromuscular programs and are becoming a lead player in this field. Our continued focus on innovative research means we are exploring new ways to use our AOC platform and expanding our horizon in terms of the types of diseases we may be able to treat. Every patient, no matter how rare their disease, deserves to have treatment options available. We are committed to realizing the full potential of our AOC platform to deliver therapeutic RNA where it is needed and provide hope for the many patients who are waiting for approved therapies. In fact, we are actively preparing for our first regulatory submission at year-end 2025 and are planning for three successive product launches starting in 2026.

An important part of our R&D and commercial launch efforts is to incorporate patient and family voices in our decision-making processes, ranging from trial design to support services. We are committed to listening, learning, and partnering with the global patient and advocacy community and have had the privilege of speaking with many advocacy leaders, organizations, and patient community members impacted by DMD44, DM1 and FSHD. Their insights are invaluable, and we take whatever steps necessary to better understand their journeys and needs to ensure we develop safe and effective therapies that will truly make a meaningful difference in their lives. As we prepare to transition from a development stage to a commercial organization, we are continuously keeping the patient community at the front of our minds. We are so grateful that their support has gotten us to this point where we can soon be launching our first drug to patients all around the world.

What do you see as the biggest challenge facing the life sciences industry today?

Rare disease drug development is associated with many unique challenges, such as small patient populations, often limited understanding of underlying disease biology, and in many cases lack of established trial endpoints and regulatory pathways, to name a few. However, in my extensive global experience in the life sciences industry, including supporting the launch of several rare disease therapies, I have seen that it is possible to overcome these inherent challenges and make a profound impact on people’s lives. The key is developing safe and well tolerated treatments that offer significant benefits, thereby creating a strong health economic argument for their value.

Patient access is another barrier that impacts many communities, especially rare disease communities. Companies must develop business strategies through a global lens, doing their part to collaborate with industry stakeholders worldwide as early as possible, including government leaders, patient advocates, regulators, healthcare technology assessment groups, payers, and others, to help support global access to new therapies.

What are the major health policy issues and themes that you are most focused on in 2025?

There are more than 7,000 known rare diseases, and yet only about 5% of them have approved treatments available. To support continued investment and advances in rare disease research, it is essential to protect critical healthcare policies and incentives, as well as continue to empower and partner with global patient advocacy organizations, which are essential to supporting and funding innovative research in the rare disease sector. Rare Disease Day is also an important time each February to collaborate with patient advocates and recognize our collective ongoing work to bring broader awareness to rare diseases. This year’s Rare Disease Day theme is “More Than You Can Imagine,” and we imagine a world where there are no barriers to treatment access and better outcomes for the millions of people around the globe who are impacted by rare diseases.

What attracted you to join EUCOPE and how can we help you achieve your business goals?

We are proud to be a member of EUCOPE as we share the same purpose: to profoundly impact the lives of people suffering from serious diseases through innovative new medicines. We were also attracted to EUCOPE’s mission to elevate the voice of and highlight the important work being done at smaller biotech companies amongst the noise of the vast life sciences industry. Our partnership is incredibly valuable and provides a great opportunity for us to connect with key industry stakeholders, including investors, clinicians, advocates, patients, and regulators, who all play a role in bringing new and innovative therapies, especially for rare diseases, from bench to bedside. These connections allow us to raise new levels of awareness about rare diseases, educate about the impact on quality of life and lack of approved treatments, and share the latest scientific research findings. Continued global collaboration across stakeholder groups is essential for us to reach our collective goal of helping patients and families impacted by rare diseases, and we look forward to ongoing support from EUCOPE in the years ahead.