Antisense Oligonucleotides Market to hit US$5,286.96 million by 2033 | Ionis Pharmaceuticals, Inc. Sarepta Therapeutics, Alnylam Pharmaceuticals, Inc

"The Global Antisense Oligonucleotides Market reached US$2,985.97 million in 2024 and is expected to reach US$5,286.96 million by 2033, growing at a CAGR of 6.5% during the forecast period 2025-2033." As per DataM intelligence research report

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United States: Recent Industry Developments

✅ In July 2025, Ionis Pharmaceuticals announced FDA clearance of its antisense drug Tofersen for SOD1-ALS.The therapy targets genetic mutations linked to amyotrophic lateral sclerosis, slowing disease progression.Commercial rollout in the U.S. is planned for Q4 2025.

✅ In June 2025, Biogen expanded its partnership with Ionis to develop next-gen antisense therapies for rare neuromuscular diseases.The $1.2 billion deal includes milestone payments and joint commercialization rights.Phase II trials for a Duchenne muscular dystrophy candidate will start in early 2026.

✅ In May 2025, Wave Life Sciences reported positive Phase Ib/IIa data for WVE-003, an antisense drug for Huntington's disease.The therapy selectively silences mutant HTT transcripts while sparing healthy alleles.FDA breakthrough therapy designation is expected later this year.

✅ In April 2025, Sarepta Therapeutics began Phase I studies on an antisense candidate for limb-girdle muscular dystrophy.The drug uses phosphorodiamidate morpholino oligomer (PMO) chemistry for targeted exon skipping.Enrollment is underway in three major U.S. clinical centers.

Japan: Recent Industry Developments

✅ In July 2025, Nippon Shinyaku initiated domestic trials of its antisense therapy for Duchenne muscular dystrophy. The candidate is designed to induce exon 53 skipping and restore partial dystrophin function. Regulatory submission in Japan is targeted for late 2027.

✅ In June 2025, Takeda Pharmaceutical partnered with Tokyo Medical University to explore antisense drugs for rare metabolic disorders. The program will focus on silencing overactive genes causing inherited enzyme deficiencies. Preclinical studies are set to begin in Q1 2026.

✅ In May 2025, Chugai Pharmaceutical filed a Japanese patent for a next-gen antisense platform with enhanced tissue penetration. The technology leverages lipid nanoparticle (LNP) delivery for improved CNS targeting. Human trials are projected to commence in 2026.

✅ In April 2025, Otsuka Pharmaceutical joined forces with Riken Institute to develop antisense oligonucleotides for psychiatric disorders. The focus will be on modulating gene expression in treatment-resistant depression.Animal studies are currently ongoing with promising early results.

Antisense Oligonucleotides Market: Drivers

The antisense oligonucleotides market is primarily driven by the growing prevalence of genetic and rare diseases, where ASOs can selectively modulate gene expression and target previously untreatable conditions. Advances in molecular biology and nucleotide chemistry are enabling the development of more stable, efficient, and targeted oligonucleotides. Increasing R&D investments by pharmaceutical and biotech companies are accelerating clinical trials and expanding the therapeutic pipeline for ASO-based drugs. Regulatory approvals for several high-profile ASO therapies are boosting market confidence and encouraging further innovation. The rise of personalized medicine and precision therapeutics is creating demand for sequence-specific treatments tailored to individual patient needs.

Market growth is also supported by the expanding applications of ASOs in oncology, neurology, cardiovascular, and metabolic disorders. Technological innovations such as chemical modifications, lipid nanoparticle delivery, and conjugation strategies are improving drug stability and cellular uptake. Collaborations between academia, biotech startups, and large pharmaceutical companies are accelerating discovery and commercialization of new ASO therapies. Government initiatives and funding programs for rare and genetic disease research are fostering innovation and adoption. Increasing awareness among healthcare providers and patients about the benefits of targeted gene therapies is further driving adoption in clinical practice.

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Segment Covered in the Antisense Oligonucleotides Market:

➥ By Drug Type: Enzyme Replacement Therapy (ERT), Hematopoietic Stem Cell Transplant (HSCT), Bone Marrow Transplantation (BMT), Others
➥ By Indication: Adult, Pediatric

Strategic Players Driving the Antisense Oligonucleotides Market Forward:

Ionis Pharmaceuticals, Inc. Sarepta Therapeutics, Alnylam Pharmaceuticals, Inc. Percheron Therapeutics, Isarna Therapeutics GmbH, Novartis AG, Jazz Pharmaceuticals, Akcea Pharma, Genzyme and Biogen among others.

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