Antisense Oligonucleotides Market

Antisense Oligonucleotides: Introduction

• Antisense oligonucleotide adoption trends are heavily influenced by their promising therapy prospects for a variety of medical conditions. Furthermore, they promote the development of treatments targeting protein targets that cannot be treated with protein therapeutics, hence driving market expansion. They target a specific faulty gene, interfere with RNA function at the cellular level, and may be silenced or manipulated, which is grabbing the interest of market participants.
• The increased demand for antisense oligonucleotides is ascribed to their ability to affect the immune system, allowing for the treatment of a wide range of autoimmune illnesses that are not treatable with currently available medications.
• When compared to other biologic medicines, it is simple to scale up commercial scale GMP production of these oligonucleotides, which encourages the use of antisense oligonucleotides.
• Furthermore, the industry is boosted by the fact that antisense oligonucleotides have few adverse effects and are easier to control than other types of medications.
• However, this is expected to change with the approval of nusinersen (Spinraza) for the treatment of spinal muscular atrophy (SMA), one of the most common hereditary causes of infant mortality with no licensed treatments.
• Even if the market performance of antisense oligonucleotides is unpredictable, as is the case with other segments of the industry, the market outlook is positively influenced by the market's leading companies and the amount of R&D expenditure.

Strong Pipeline of Antisense Oligonucleotides is expected to drive the Antisense Oligonucleotides Market

• A strong pipeline of antisense oligonucleotides, as well as increasing focus on a high number of product approvals and commercialization, are expected to grow the size of the worldwide antisense oligonucleotide business. Furthermore, major improvements in the therapeutic and clinical use of antisense oligonucleotides are adding to the global market's growth. However, the global antisense oligonucleotide business faces significant obstacles due to oligonucleotide diversity, delivery, and regulatory complexity. For instance, in December 2024, U.S. FDA approved Tryngolza (also known as Olezarsen) for the treatment of adults with familial chylomicronemia syndrome (FCS).
• Similarly, various other antisense oligonucleotides were approved. For instance, in June 2024, Geron Corporation announced that the U.S. Food and Drug Administration (FDA) has approved RYTELO (imetelstat) for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent (TD) anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).

Increased Research and Development Investment is Attributed to Drive Overall Antisense Oligonucleotides Market

• Biopharmaceutical companies' continual efforts in research and development are important to the ASO market's growth. For example, Biogen funded $2.5 billion in R&D in 2022, allowing for the development of new applications for ASOs, particularly in uncommon genetic illnesses and cancer. The ongoing partnership between pharmaceutical corporations and academic institutions is accelerating innovation in ASO treatments, broadening their therapeutic spectrum and propelling the market forward.
• Also, in March 2024, Celanese Corporation, and Secarna Pharmaceuticals GmbH & Co. KG, announced a research collaboration for the development of long-acting implants that deliver antisense oligonucleotides (ASOs).

By Drug Type, the Nusinersen segment accounted for the largest share of the Antisense Oligonucleotides industry

• The Nusinersen drug type holds the largest market share. Nusinersen, is commercialized as Spinraza, which is used for the treatment of spinal muscular atrophy (SMA) and has achieved a commercial success. Moreover, growing research activities related to the launch of higher potential of antisense oligonucleotides has further resulted in the growth of the market segment. For instance, in January 2025, the U.S. FDA has accepted Biogen’s supplemental new drug application (sNDA) for a higher, potentially more efficacious dose regimen of nusinersen (Spinraza) for patients with spinal muscular atrophy (SMA). The company also reported that the European Medicines Agency (EMA) has validated the application for the higher dose as well.
• The current approved regimen for nusinersen is four 12 mg doses administered over 60 days, followed by maintenance dosing every four months. Both therapies are injected directly into the lumbar spine.

North America to Lead the Antisense Oligonucleotides Market

• North America dominates the antisense oligonucleotides market, owing to its strong product pipeline and an increasing number of U.S. FDA approvals across the region, which is contributing to the region’s marker share of 45.2% in 2024. For instance, in January 2024, Vanda Pharmaceuticals Inc., announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application to evaluate VCA-894A for the treatment of a patient with Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within the IGHMBP2 gene.
• Effective treatments for uncommon diseases require faster drug discovery methodologies, notwithstanding tremendous progress. Therapeutic antisense oligonucleotides can influence target gene expression with great specificity via a variety of methods determined by base sequences and chemical modifications, and have demonstrated success in clinical studies for a few rare neurological disorders.

Key Players Operating in the Global Antisense Oligonucleotides Market

The major players operating in the global market are focusing on advancements in research and development to meet the rising demand for antisense oligonucleotides. Moreover, manufacturers are entering into partnerships for the development of innovative products.

Key players operating in the global antisense oligonucleotides market include:

• Ionis Pharmaceuticals, Inc.
• Biogen
• Alnylam Pharmaceuticals, Inc.
• Sarepta Therapeutics
• Arrowhead Pharmaceuticals, Inc.
• Atlantic Pharmaceuticals, Inc.
• Antisense Therapeutics Limited
• Bio-Path Holdings, Inc.
• Gene Signal International SA
• GlaxoSmithKline plc
• Other Prominent Players

Antisense Oligonucleotides Market Segmentation

• By Drug Type:
  • Imetelstat
  • Olezarsen
  • Eteplirsen
  • Nusinersen
  • Inotersen
  • Volanesorsen
  • Others (Golodirsen, Viltolarsen, Late Phase Drugs, etc.)
• By Indication:
  • Ocular Diseases
  • Cancer
  • Diabetes
  • Amyotrophic Lateral Sclerosis (ALS)
  • Duchenne Muscular Dystrophy
  • Others (Spinal Muscular Atrophy, etc.)
• By Route of Administration:
  • Parenteral
  • Oral
• By Distribution Channel:
  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies
• By Region:
  • North America
    • U.S.
    • Canada
  • Europe
    • Germany
    • France
    • U.K.
    • Italy
    • Spain
    • Rest of Europe
  • Asia Pacific
    • China
    • Japan
    • India
    • Australia & New Zealand
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East & Africa
    • GCC Countries
    • South Africa
    • Rest of Middle East & Africa