Avidity Biosciences, Inc.

Avidity is expanding beyond rare neuromuscular disorders, leveraging our proprietary AOC platform to address the root causes of genetic heart diseases in precision #cardiology. With continued AOC innovations, we're pushing the boundaries of RNA delivery to transform care for people with serious and rare diseases. Learn more: https://lnkd.in/ebDQ-Xvz

We are honored to be recognized as a finalist in two categories for the 2025 Citeline Awards: Excellence in Rare Disease Drug Development and Clinical Trial Result of the Year. This past year has been an important one for our team, as we’ve made significant strides in advancing our clinical-stage programs for rare neuromuscular diseases, including #DM1, #DMD, and #FSHD. A key highlight was the presentation of initial data from our Phase I/II FORTITUDE trial in FSHD, which provided early insights into the safety and exploratory endpoints of our investigational therapy, marking an important step in its continued development for the FSHD community. This recognition would not be possible without the dedication of our team, along with the support and partnership of patients, families, and the broader rare disease community. We look forward to being recognized alongside other leaders in the field at the awards ceremony next month!

This August, our very own John Moriarty will be lacing up his boots for the Timmerman Traverse, joining life science leaders across the industry on a mission rooted in impact supporting Life Science Cares and their work to disrupt the cycle of poverty and inequality in our communities. As a longtime partner of Life Science Cares, Inc., we're proud to support John's endeavor and his commitment to the cause. At our core, we believe that meaningful progress, whether on a mountain trail or in the lab, comes from perseverance and shared purpose while never losing sight of the communities we serve. #TimmermanTraverse2025 #LifeScienceCares

Join our President and CEO Sarah Boyce and CMO Steve Hughes, MD, today at 3:00pm ET for a #firesidechat during the Needham Healthcare Conference to learn more about the exciting progress we are making with our three clinical programs for #DM1, #DMD44 and #FSHD and our expansion into precision #cardiology. To view the webcast, visit: https://lnkd.in/grNtBgD

We are pleased to announce that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation to our AOC in clinical development for myotonic dystrophy type 1 (#DM1). This will be the first-ever investigational treatment for DM1 to receive Orphan Drug designation in Japan. DM1 is an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. We continue to plan for multiple key milestones this year and accelerate our commercialization preparations to bring a treatment option to people living with DM1 as quickly as possible. Learn more in our press release: https://lnkd.in/eCePfkGf

At Avidity, we are advancing muscle-targeted RNA therapeutics for Duchenne muscular dystrophy (DMD) through our AOC platform. We are excited to take the next step in our journey, with plans to submit our first BLA submission for an accelerated approval to the FDA this year. As we continue to advance our research, we remain focused on the #Duchenne community and the promise of our approach to make a meaningful impact. Read more in Endpoints News: https://lnkd.in/eUE79Gc5

We were honored to welcome members of The Akari Foundation to Avidity’s headquarters last month in San Diego. It was a great opportunity to connect, share our mission, and learn more about the incredible work Akari is doing to support the Duchenne community. Collaborations like this help strengthen our commitment to diversity and inclusion efforts to advance science and provide valuable resources to families affected by rare diseases. We look forward to continuing these important conversations!

Last week during the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, we had the privilege of hosting an Industry Forum Breakfast focused on the potential of antibody oligonucleotide conjugates (AOCs™) in rare neuromuscular diseases. Our CSO, Dr. Mike Flanagan, led the discussion on how Avidity is advancing clinical development for #DM1, #FSHD, and #DMD with our AOC platform. A huge thank you to everyone who attended, visited our booth during the #MDAConference, and engaged with our team—we deeply appreciate your enthusiasm and collaboration. Learn more about our AOC platform: https://lnkd.in/eNxaEU2X

Today we announced the completion of enrollment in the biomarker cohort in the Phase 1/2 FORTITUDE™ clinical trial of our AOC in development for facioscapulohumeral muscular dystrophy (FSHD). #FSHD is a rare, progressive, and variable hereditary muscle-weakening condition characterized by life-long, relentless loss of muscle function, significant pain, fatigue, and progressive disability, with no current treatments available. Completing enrollment in the biomarker cohort marks an important milestone as we pursue a potential accelerated approval path in the U.S., bringing hope to the FSHD community as we work to deliver the first approved treatment for this devastating neuromuscular disease.  For more information, see our press release: https://lnkd.in/eF-gHSqy

We're excited to join Parent Project Muscular Dystrophy for a webinar today at 2:00pm ET, where we will discuss topline data from our Phase 1/2 EXPLORE44® trial evaluating the safety and efficacy of our investigational therapy for people living with Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44). These data were presented during the #MDAconference earlier this month. Join us for this important discussion and take part in a live Q&A with our team: https://lnkd.in/eXju9auT