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Here are 1-3 brief analogies for Avidity Biosciences (symbol: RNA):

1. Like **Seagen** (now part of Pfizer), but instead of using antibodies to deliver chemotherapy, Avidity delivers genetic medicines (RNA) to specific cells.
2. Imagine **Alnylam Pharmaceuticals**, a pioneer in RNA interference, but with a highly specialized antibody "targeting system" to deliver their genetic therapies precisely to affected cells.
3. Think of it as a more precise version of the genetic medicine approach popularized by companies like **Moderna**, using antibodies to ensure RNA therapies reach only the intended cells.

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• AOC 1001: An investigational therapeutic designed to treat Myotonic Dystrophy Type 1 (DM1), a rare inherited neuromuscular disease.
• AOC 1020: An investigational therapeutic being developed for Facioscapulohumeral Muscular Dystrophy (FSHD), a progressive genetic muscle disorder.
• AOC 1044: An investigational therapeutic targeting Duchenne Muscular Dystrophy (DMD) patients who have mutations amenable to exon 44 skipping.

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Avidity Biosciences (NASDAQ: RNA) is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs).

As a clinical-stage company, Avidity Biosciences does not currently have any commercial products on the market, and therefore, does not have "major customers" in the traditional sense of direct product sales to other companies or individuals.

However, Avidity Biosciences does engage in strategic collaborations that provide funding and support for its research and development efforts. The most significant of these, which could be considered a "customer" for its technology and pipeline assets in a broader sense, is:

• Eli Lilly and Company (NYSE: LLY)

In December 2020, Avidity entered into a research collaboration and exclusive license agreement with Eli Lilly and Company to design, develop, and commercialize AOCs for immunology and other indications outside of muscle. This collaboration involves upfront payments, research funding, and potential milestone payments and royalties.

Ultimately, if Avidity's drug candidates receive regulatory approval in the future, they would be prescribed by healthcare professionals to treat individuals suffering from rare muscle diseases, such as myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne muscular dystrophy (DMD). The distribution of such approved therapies would typically occur through established pharmaceutical channels, involving pharmaceutical distributors, hospitals, and pharmacies, with costs often covered by healthcare payers (insurance companies or government programs).

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Sarah Boyce, President & CEO

Sarah Boyce joined Avidity Biosciences as President and Chief Executive Officer and a member of the Board of Directors in October 2019. With over 25 years of global leadership experience in the pharmaceutical and biopharmaceutical industries, she has been instrumental in transitioning Avidity from a research-focused entity to a clinical-stage biopharmaceutical firm, driving a Series C funding round and an IPO. Prior to Avidity, Ms. Boyce served as President and a board member of Akcea Therapeutics, where she led the commercialization of rare disease products. She also held executive roles in business development and global operations at leading life sciences companies including Ionis Pharmaceuticals, Forest Laboratories, Alexion Pharmaceuticals, Novartis Oncology, and Roche. Her experience includes the RNA space and rare diseases, and she has successfully brought innovative therapies like Tegsedi®, Waylivra®, Soliris®, Gleevec®, and Tasigna® to patients. Ms. Boyce currently serves as a member of the board of directors at Contineum Therapeutics, Inc., and Abcuro, Inc.

Michael MacLean, Chief Financial and Chief Business Officer

Michael MacLean joined Avidity Biosciences as Chief Financial Officer in May 2020, and his role was expanded to include Chief Business Officer responsibilities in April 2022. He brings decades of strategic financial leadership within life sciences and other industries, with deep experience in funding biotechnology companies to advance novel therapies in the rare and orphan disease space. Before joining Avidity, Mr. MacLean served as Chief Financial Officer of Akcea Therapeutics, Inc., where he led the buildout of Akcea's financial and commercial infrastructure. He also held the position of Chief Financial Officer at PureTech Health, plc, and Chief Accounting Officer at Biogen Inc., where he led the company's worldwide finance operations. Mr. MacLean currently serves as a member of the board of directors at Verve Therapeutics.

Arthur A. Levin, Chief Scientific Officer

Arthur A. Levin previously served as Executive Vice President of Research and Development at miRagen Therapeutics. He held senior drug development roles at Ionis (formerly Isis) Pharmaceuticals and Santaris Pharma. Dr. Levin has played key roles in the development of numerous oligonucleotides, including the first approved antisense drugs and the first microRNA-targeted therapeutic in clinical trials, bringing three decades of experience in drug development from discovery through drug registration in both large pharma and biotech companies.

Steve Hughes, MD, Chief Medical Officer

Dr. Steve Hughes joined Avidity Biosciences in February 2022, bringing over 20 years of experience in building and leading clinical development and medical affairs teams. He has contributed to over 50 clinical trials for more than 25 drugs across various therapeutic areas, including cardiovascular, neurology, and several rare diseases. Prior to Avidity, Dr. Hughes was with Arcturus Therapeutics, where he continues to serve as a strategic clinical advisor.

Teresa McCarthy, Chief Human Resources Officer

Teresa McCarthy joined Avidity Biosciences in August 2020 and is responsible for developing and executing human resource strategies in support of the company's overall business plan. She brings decades of experience as an organizational development and human resources consultant in the life science industry, having worked with biotech and healthcare companies at all stages of development through her consulting firm, McCarthy Consultants Inc., and her long-term partnership with The Leadership Edge, Inc.

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1. Clinical Trial Setbacks and Regulatory Hurdles

   As a clinical-stage biopharmaceutical company, Avidity's valuation and future success are heavily dependent on the successful progression of its Antibody Oligonucleotide Conjugates (AOCs™) platform through clinical trials and subsequent regulatory approvals. Any delays in planned Biologics License Application (BLA) submissions, such as for del-zota beyond 2026, or disappointing data from late-stage trials, like the Phase 3 HARBOR™ trial for del-desiran expected in Q2 2026, could severely impact the company's stock performance. Furthermore, unexpected adverse side effects in late-stage trials or changes in regulatory guidance could halt or significantly delay the path to market for its candidates.

2. High Cash Burn and Unprofitability for SpinCo

   Avidity Biosciences has been in a high-burn, pre-commercial phase, reporting significant net losses as it heavily invests in research and development to advance its pipeline. For the 2025 fiscal year, analysts project a net loss of approximately $-675.4 million. While the recent acquisition of a significant portion of Avidity's assets by Novartis for approximately $12 billion fundamentally alters the financial outlook for the acquired assets, the remaining early-stage precision cardiology programs, which are being spun out into a new public entity (SpinCo), will face the challenge of managing their own financial trajectory without the immediate backing of the full acquisition valuation. The substantial R&D spend and ongoing losses for these independent programs represent a continued financial risk, as profitability may be years away.

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A clear emerging threat for Avidity Biosciences is the advancement of Dyne Therapeutics and its FORCE™ platform. Dyne Therapeutics is developing a similar class of targeted oligonucleotide therapeutics, also leveraging an antibody to deliver RNA to specific cell types, specifically for rare muscle diseases. This directly competes with Avidity's Antibody Oligonucleotide Conjugates (AOCs) platform and their lead programs.

• Dyne's lead program, DYNE-101 for Myotonic Dystrophy Type 1 (DM1), directly competes with Avidity's AOC 1001 for DM1. Both aim to target the DMPK gene to reduce toxic RNA and improve muscle function. Dyne has reported positive preliminary clinical data from its ACHIEVE trial.
• Dyne is also developing DYNE-251 for Duchenne Muscular Dystrophy (DMD) amenable to exon 51 skipping, which could compete with Avidity's developing DMD programs (e.g., AOC 1020).
• Furthermore, Dyne has DYNE-301 for Facioscapulohumeral Muscular Dystrophy (FSHD), a direct competitor to Avidity's AOC 1044 for FSHD.

The head-to-head competition with Dyne Therapeutics, utilizing a highly similar technology platform for the same rare muscle disease indications, represents a clear and emerging threat to Avidity's market position and pipeline success.

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Avidity Biosciences is focused on developing therapies for rare neuromuscular diseases through its Antibody Oligonucleotide Conjugate (AOC) platform. The addressable markets for their main product candidates are as follows:

• Del-desiran (AOC 1001) for Myotonic Dystrophy Type 1 (DM1): The estimated patient population for DM1 is approximately 80,000 across the United States and Europe. Another estimate places the patient population at about 40,000 in the U.S. and a similar number in Europe. Analysts have projected peak sales for del-desiran to reach $4.3 billion.
• Del-brax (AOC 1020) for Facioscapulohumeral Muscular Dystrophy (FSHD): The patient population for FSHD is estimated to be between 45,000 and 87,000 in the United States and Europe. Another source suggests around 30,000 patients in the U.S. and a similar number in Europe. The FSHD treatment market in the U.S. and EU is projected to grow to $61.4 million by 2035 from $32.2 million in 2024. Other estimates for the FSHD market indicate a potential worth of $4-5 billion. In 2021, the total market size for FSHD in the United States was reported as $19.83 million, and in Japan, it was $2.39 million. Globally (across the 7 major markets), the total FSHD market size was $25.55 million in 2021. BMO Capital Markets analysts have predicted peak sales for del-brax of $3.2 billion.
• Del-zota (AOC 1044) for Duchenne Muscular Dystrophy (DMD) exon 44 skipping: Del-zota targets a specific subset of DMD patients, those amenable to exon 44 skipping, which accounts for approximately 6-7% of all DMD cases in the U.S. The broader global DMD market is expected to reach $7.4 billion by the end of 2034. Projected U.S. sales for del-zota are approximately $400 million. BMO Capital Markets analysts have predicted peak sales for this candidate to be $420 million.

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Avidity Biosciences (RNA) is poised for significant future revenue growth over the next two to three years, driven primarily by the advancement and commercialization of its Antibody Oligonucleotide Conjugates (AOCs) pipeline, as well as continued strategic collaborations.

Here are 3-5 expected drivers of Avidity Biosciences' future revenue growth:

1. Commercialization and Launch of Del-zota (DMD44): Avidity Biosciences anticipates submitting a Biologics License Application (BLA) for del-zota, its treatment for Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping (DMD44), by the end of 2025. A potential commercial launch in the U.S. could follow in 2026, marking the company's first potential product on the market.
2. Commercialization and Launch of Delpacibart Braxlosiran (del-brax, FSHD): The company plans to submit a BLA for accelerated approval of delpacibart braxlosiran (del-brax) for the treatment of facioscapulohumeral muscular dystrophy (FSHD) in the second half of 2026. Positive Phase 1/2 data and alignment with the FDA on approval pathways support this driver.
3. Advancement and Potential Commercialization of Del-desiran (DM1): Del-desiran, Avidity's therapy for myotonic dystrophy type 1 (DM1), is currently in the global Phase III HARBOR trial and the MARINA-OLE trial. Successful progression and eventual commercialization of this program represent another significant future revenue stream.
4. Continued and Expanded Collaboration Agreements: Avidity Biosciences currently generates revenue through research collaboration and license partnerships, such as with Bristol Myers Squibb. These agreements provide upfront payments, milestone payments, and potential future royalties, contributing to revenue growth as pipeline programs advance and new partnerships may be established.

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Share Issuance

• Avidity Biosciences closed its initial public offering (IPO) on June 16, 2020, issuing 16,560,000 shares at $18.00 per share, which generated approximately $298.1 million in gross proceeds.
• In September 2025, Avidity Biosciences completed an upsized public offering of 17,250,000 shares of common stock at $40.00 per share, resulting in gross proceeds of $690.0 million and net proceeds of approximately $651.4 million. The company intends to use these funds to advance its late-stage clinical programs, build commercial inventory, expand infrastructure, and further research and development of its AOC™ platform.
• Avidity also generated $185.5 million in net proceeds from the sale of 5,646,583 shares through its at-the-market (ATM) offering in the second and third quarters of 2025.

Inbound Investments

• In October 2025, Novartis announced an agreement to acquire Avidity Biosciences for approximately $12 billion in cash, with Avidity shareholders receiving $72.00 per share. The acquisition, anticipated to close in the first half of 2026, is contingent on Avidity separating its early-stage precision cardiology programs into a new public company, "SpinCo".
• The "SpinCo" entity, which will hold Avidity's early-stage precision cardiology programs, is expected to be funded with $270 million.

Outbound Investments

• As part of the acquisition by Novartis, Avidity Biosciences plans to separate its early-stage precision cardiology programs into a new public company, "SpinCo", prior to the closing of the merger. Avidity shareholders are expected to receive one share of SpinCo for every ten shares of Avidity held.

Capital Expenditures

• Avidity Biosciences reported capital expenditures of approximately $-12.72 million in 2025, $-7.07 million in 2024, and $-4.23 million in 2023.
• The company has entered into minimum purchase obligations for commercial manufacturing totaling $621.6 million over the period of 2026–2028.
• Proceeds from the September 2025 public offering are designated, in part, for expanding commercial infrastructure and advancing research and development associated with its AOC platform.