Towards Healthcare

Small Nucleic Acid Drug Companies and Market Trends Analysis

Date : 13 October 2025

Top Companies in the Small Nucleic Acid Drug Market

Small Nucleic Acid Drug Market Companies

  • Ionis Pharmaceuticals
  • Alnylam Pharmaceuticals
  • Sarepta Therapeutics
  • Biogen
  • Moderna
  • Novartis
  • Regeneron Pharmaceuticals
  • Arrowhead Pharmaceuticals
  • Silence Therapeutics
  • Dicerna (Novo Nordisk)
  • Wave Life Sciences
  • Stoke Therapeutics
  • Arbutus Biopharma
  • Apellis Pharmaceuticals
  • OliX Pharmaceuticals
  • Sirnaomics
  • Genevant Sciences
  • BioNTech
  • Exicure
  • Roche

Market Growth

The global small nucleic acid drug market size is calculated at USD 5.94 billion in 2024, grew to USD 7.06 billion in 2025, and is projected to reach around USD 33.28 billion by 2034. The market is expanding at a CAGR of 18.84% between 2025 and 2034.

Increasing Collaboration: Major players collaborate to share proprietary technologies, expertise, and infrastructure to expand their product pipelines. 

  • In April 2025, Creyon Bio, Inc. announced a collaboration with Eli Lilly and Company to focus on the discovery, development, and commercialization of novel RNA-targeted oligonucleotide therapies. Creyon will leverage its AI-powered Oligo Engineering Engine for the development.

FDA Approval: The U.S. Food and Drug Administration (FDA) provides market authorization for various SNADs based on their preclinical and clinical trial data.

  • In November 2024, HuidaGene Therapeutics announced that the FDA cleared its investigational new drug application (IND) for its HG202, a clinical-stage Cas 13-based RNA editing therapeutic candidate. HG202 will be tested in clinical trials in patients with neovascular age-related macular degeneration (nAMD).   

Value Chain Analysis - Small Nucleic Acid Drug Market

R&D

The research activities for SNADs include designing and evaluating novel SNADs for diverse diseases. Scientists also develop novel drug delivery systems for the targeted delivery of SNADs.

Key Players: Axolabs GmbH, AstraZeneca, and Ionis Pharmaceuticals

Clinical Trials & Regulatory Approvals

Clinical trials are conducted to either find expanded applications for existing SNADs or study the safety and efficacy of novel SNADs against a particular disease.

Key Players: Novartis Pharmaceuticals, Isarna Therapeutics GmbH, Bio-Path Holdings, Inc.

Distribution to Hospitals, Pharmacies

The final drug product of SNADs is distributed to hospitals and specialty or retail pharmacies by manufacturers or wholesalers.

Patient Support & Services

Healthcare professionals provide personalized care to patients by prescribing SNADs. They also educate patients about the dosage regimen and therapeutic benefits of SNADs.

Latest Announcement by Industry Leaders

Daniel J. Siegwart, Co-founder & Chief Scientific Advisor at SignifyBio, commented that their modular platform, Signal Peptide Engineered Nucleic acid Design (SEND), offers an unprecedented opportunity to engineer personalized protein therapeutics directly within the body, addressing long-standing challenges in drug production, delivery, and efficacy. The company lays the groundwork for a new generation of precision medicine across a wide range of chronic and genetic disorders with its versatility and adaptability.

Recent Developments in the Small Nucleic Acid Drug Market

  • In July 2025, researchers from the University of Basel revealed that slowing down the intracellular transport of RNA-based drugs, like ASO, can significantly enhance their effectiveness. They used the CRISPR/Cas9 technology to identify factors that significantly influence ASO activity.
  • In November 2024, researchers from Shinshu University determined the role of osteogenic CpG oligodeoxynucleotide (iSN40) in inhibiting osteoclastogenesis. They found that iSN40 can modulate both bone formation and resorption, highlighting its application in the treatment of osteoporosis.

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