Avidity Biosciences, Inc.

Company overview

• Avidity Biosciences is a biopharmaceutical company developing Antibody Oligonucleotide Conjugates (AOCs), a class of RNA therapeutics that combine monoclonal antibodies with oligonucleotides to target diseases that are difficult to treat with conventional approaches.
• As of February 13, 2026, the company had 511 full-time employees, including 111 with a Ph.D. or M.D. degree.
• No product revenue has been generated to date.

Technology and platform

• AOC platform: targeted antibody delivery of oligonucleotides (e.g., siRNAs, PMOs) to specific tissues and cell types, initially focused on muscle with plans to expand to other tissues.
• Mechanism: an antibody (commonly targeting TfR1) binds a cell surface protein and delivers the attached oligonucleotide to target cells, enabling modulation of RNA and downstream disease pathways.
• Oligonucleotide types: small interfering RNAs (siRNAs) and phosphorodiamidate morpholino oligomers (PMOs).
• Key advantages: targeted delivery to non-hepatic tissues, potential for infrequent dosing, scalable manufacturing using established monoclonal antibody and oligonucleotide production methods, and the ability to reuse a single monoclonal antibody across multiple programs.

Development pipeline and programs

• Del-desiran (AOC 1001) — Duchenne muscular dystrophy type 1 (DM1)

  • Target: DMPK mRNA via TfR1-targeted siRNA (siDMPK.19).
  • Stage: Global Phase 3 HARBOR trial in DM1 with an ongoing HARBOR-OLE extension.
  • Regulatory designations: FDA Breakthrough Therapy, Orphan Drug, and Fast Track in the U.S.; Orphan designation in the EU and Japan.
  • Recent activity: 54-week data readout anticipated in 2026; protocol amendments submitted to FDA regarding 54-week data. Earlier MARINA-OLE data were published and support possible disease modification signals.

• Del-brax (AOC 1020) — Facioscapulohumeral muscular dystrophy (FSHD)

  • Target: DUX4 mRNA via TfR1-targeted siRNA (siDUX4).
  • Stage: Global Phase 1/2 FORTITUDE program, including a biomarker cohort (FORTITUDE Biomarker Cohort) and a planned Phase 3 global FORTITUDE-3 trial.
  • Regulatory designations: Orphan designation and FDA Fast Track for FSHD.
  • Timeline: Phase 3 readout planned for 2028; biomarker cohort topline data anticipated in Q2 2026.

• Del-zota (AOC 1044) — Duchenne muscular dystrophy with exon 44 skipping (DMD44)

  • Target: PMO-based exon-skipping PMO conjugated to a TfR1 AOC to induce exon 44 skipping in dystrophin mRNA.
  • Stage: Phase 2 EXPLORE44-OLE (Open-Label Extension) with a Managed Access Program announced in the U.S. in November 2025.
  • Regulatory designations: Orphan and FDA Breakthrough Therapy; FDA Fast Track and Rare Pediatric Disease designations.
  • Regulatory plan: BLA submission planned for 2026 for potential accelerated approval.

• Cross-program and earlier-stage assets

  • The three neuromuscular programs above are the company’s first wholly owned neuromuscular and precision cardiology AOC programs.
  • Early-stage cardiology programs include AOC 1086 (PLN cardiomyopathy) and AOC 1072 (PRKAG2 syndrome).
  • The company has additional research collaborations in immunology and other indications.

Strategic collaborations and economics

• Bristol Myers Squibb (BMS)

  • Agreement: November 2023 collaboration to develop up to five cardiovascular targets using the AOC platform.
  • Financial terms: approximately $100 million upfront (about $60 million cash under the collaboration and roughly $40 million via stock purchase).
  • Potential payments: up to $1.35 billion in R&D milestones and up to $825 million in commercial milestones, plus tiered royalties on net sales (high single digits to low double digits).

• Eli Lilly

  • Agreement: April 2019 collaboration; Lilly funds preclinical research and discovery and leads clinical development, regulatory approval, and commercialization for Lilly AOCs. Lilly has an exclusive worldwide license to up to six mRNA targets.
  • Financial terms: $20.0 million upfront; eligible for up to $60.0 million in development milestones, up to $140.0 million in regulatory milestones, and up to $205.0 million in commercialization milestones per target; tiered royalties on net sales.

• MyoKardia / BMS

  • Timeline: December 2020 collaboration with MyoKardia to explore cardiac AOCs. In November 2023, BMS exercised its option to negotiate a license agreement covering cardiovascular AOCs. The earlier MyoKardia collaboration was terminated in favor of the broader BMS agreement.

• Collaboration considerations

  • Lilly and BMS may terminate their agreements under certain conditions. These partnerships are a core component of the company’s development strategy and funding for selected candidates.
  • The company may seek additional collaborations, licenses, or strategic transactions that could involve relinquishing certain rights or revenue streams.

Manufacturing and supply

• The company does not own manufacturing facilities and relies on third-party manufacturers and suppliers for antibodies, oligonucleotides, and linkers.
• Lonza Manufacturing Services Agreement (MSA)
  • Effective August 2025: Lonza will manufacture drug substance and drug product for potential commercial use.
  • Commitments: rolling forecasts with binding minimum purchases of approximately $621.6 million over 2026–2028, subject to currency and price adjustments.
  • Term: seven-year agreement ending August 1, 2032.
• Third-party manufacturing introduces risks of supply disruption, quality control issues, and regulatory compliance challenges.

Intellectual property

• Portfolio summary (as of December 31, 2025):
  • 49 issued U.S. patents and 51 pending U.S. patent applications.
  • Exclusive licenses from University of Alberta and Fred Hutchinson Cancer Center; additional licensed IP from GenAhead Bio Inc.
  • Platform IP: 40 patent families covering core AOC components; 46 issued U.S. patents and 46 granted foreign patents; 51 pending U.S. applications; 164 pending foreign applications in multiple jurisdictions.
• Targeted product IP: separate patent families for Del-desiran, Del-brax, Del-zota, AOC 1072, and AOC 1086, with various expiration timelines and potential patent term extensions.
• IP strategy: protect antibodies, oligonucleotide structures and sequences, AOC structures, and manufacturing methods.
• Risks: potential patent challenges, changes in patent law, and competing technologies.

Regulation and development path

• Regulatory approach follows standard U.S. and international processes for biologics and modalities adjacent to gene therapy.
• The company is pursuing accelerated approval pathways and orphan designations for certain programs.
• Ongoing global Phase 3 and Phase 2/3 trials and Open-Label Extensions are in progress, with planned regulatory milestones and potential post-approval obligations.

Financial and corporate highlights (selected, 2025)

• Revenue and profitability
  • No product revenue to date.
  • Net losses: $684.6 million (year ended December 31, 2025); $322.3 million (2024); $212.2 million (2023).
  • Accumulated deficit: approximately $1.6 billion as of December 31, 2025.
• Cash and financing
  • The company believes existing cash, cash equivalents, and marketable securities could fund operations for at least the next 12 months.
  • 2025 equity activity: 5,646,583 shares sold under the 2024 Sales Agreement, yielding net proceeds of $185.5 million.
• M&A and corporate actions
  • October 25, 2025: entered into a Merger Agreement with Novartis AG and a Separation and Distribution Agreement to separate SpinCo (early-stage precision cardiology assets). SpinCo will receive one SpinCo common stock for every ten shares of company common stock held.
  • Expected timing: Merger, Distribution, and related transactions expected to close in the first half of 2026.
  • Termination fees: termination of the Merger could trigger a $450 million fee; a reverse termination fee of $600 million could be payable by the other party under certain circumstances.
  • Distribution Record Date: February 12, 2026; the Distribution could occur prior to the Effective Time with SpinCo becoming an independent public company.

Operational focus

• Advancing three core AOC programs in neuromuscular diseases (DM1, DMD44, FSHD) while expanding into precision cardiology and other tissues.
• Supporting late-stage development and potential commercialization through a substantial IP portfolio and outsourced manufacturing arrangements.
• Pursuing a major corporate restructuring (Merger with Novartis and SpinCo separation) with an anticipated near-term close in 2026 that will affect governance, cash flows, and asset ownership.
• Near-term financials indicate continued loss-making operations with no product revenue, supported by financing activities and potential collaboration milestones.