Global Oligonucleotide Therapeutics Market Reaching $17.70 Billion by 2030

Oligonucleotide Therapeutics Market by Molecule (Nusinersen, Vutrisiran, Inclisiran, Eteplisiran), Type (ASO, siRNA, Others), Disease Type (Neurology, Rare Diseases, Others), RoA (SC, Intrathecal, IV, Others), and Region - Global Forecast to 2030
The global oligonucleotide therapeutics market is entering a decisive growth phase as RNA-targeting therapies gain broader clinical and commercial acceptance across major disease areas. Valued at $5.92 billion in 2024 and rising to $7.19 billion in 2025, the market is projected to expand at a robust CAGR of 19.7% from 2025 to 2030, reaching a forecasted valuation of $17.70 billion by the end of the decade.

This growth underscores a fundamental shift in how pharmaceutical and biotechnology companies are approaching precision medicine. What is driving this surge? The answer lies in the accelerating adoption of small interfering RNA (siRNA) and antisense oligonucleotide (ASO) therapies, supported by major advances in delivery platforms such as GalNAc conjugates and lipid nanoparticles. These innovations are enabling higher potency, improved stability, and enhanced tissue selectivity-key attributes that are reshaping therapeutic development strategies worldwide.

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RNA-Based Therapies Move from Promise to Commercial Reality

The market's upward trajectory is fueled by a maturing pipeline of late-stage clinical programs and increasing approvals of oligonucleotide-based drugs. Who is driving demand? Large pharmaceutical companies and emerging biotech innovators alike are scaling procurement of high-purity, GMP-grade therapeutic oligonucleotides, including custom-designed sequences, modified backbones, and conjugated formats to optimize dosing durability and targeting efficiency.

At the same time, how is innovation expanding the market scope? Breakthroughs in gene editing are creating incremental demand for advanced oligonucleotide capabilities, particularly in guide RNA design and optimization. As molecular decision-making becomes central to drug development, the need for precision, consistency, and regulatory-grade quality continues to intensify.

Trends and Disruptions Reshaping Downstream Demand

A rapid shift toward RNA-based drug modalities is transforming the pharmaceutical landscape. As programs progress from clinical development toward commercialization, why does quality matter more than ever? End users are prioritizing ultra-low impurity profiles, robust analytical characterization, and strict compliance with evolving regulatory expectations. This transition is increasing utilization of sophisticated synthesis technologies and advanced analytical workflows across the value chain.

Key Market Dynamics: Drivers, Restraints, Opportunities, and Challenges

Driver : Expanding Therapeutic and Diagnostic Applications:

The increasing use of synthesized oligonucleotides in both therapeutics and diagnostics remains a primary growth engine. Beyond ASO and siRNA drugs, diagnostics rely heavily on oligonucleotides for PCR, qPCR, next-generation sequencing (NGS), and molecular assays. The rise of precision medicine and liquid biopsy procedures is further accelerating volume demand across development and commercial settings.

Restraint: Technical and Manufacturing Complexities:

Despite strong momentum, what is limiting faster adoption? Therapeutic oligonucleotides require precise sequence design, complex chemical modifications, and stringent purity control. Challenges in targeted delivery and endosomal escape increase development risk, cost, and timelines compared to conventional small molecules.

Opportunity: Rising R&D Investment in Emerging Economies: Where is the next wave of growth coming from?

Expanding R&D investments across Asia and other emerging markets are opening new opportunities. Government-backed grants, infrastructure development, and local GMP capabilities are strengthening regional ecosystems, reducing reliance on Western suppliers, and unlocking new demand centers.

Challenge: Lack of Harmonized Global Regulations:A persistent challenge remains the absence of standardized regulatory frameworks. As oligonucleotides sit between small molecules and biologics, variations in CMC requirements, impurity profiling, and analytical expectations across regions create uncertainty for developers scaling toward commercialization.

A Complex, End-to-End Market Ecosystem

The oligonucleotide therapeutics market spans the full supply chain-from raw materials to commercial drug production. Upstream activities include suppliers of phosphoramidites, solid supports (CPG), reagents, and high-purity solvents, alongside synthesizer and automation providers enabling scalable manufacturing across laboratory, pilot, and GMP environments.

Segment Insights Highlight Commercial Leadership

By Molecule: In 2024, nusinersen (SPINRAZA) emerged as the leading segment. Approved in 2016 for spinal muscular atrophy (SMA), the therapy benefits from strong physician familiarity and established reimbursement. Biogen reported approximately US$1.5 billion in global SPINRAZA revenue in 2024, sustaining demand despite newer entrants.

By Disease Type: Neurological disorders accounted for the largest market share in 2024, reflecting the early success of oligonucleotide therapies in neurology and their suitability for long-term, specialist-driven treatment pathways, including SMA and hereditary transthyretin amyloidosis with polyneuropathy.

By Route of Administration: Subcutaneous delivery dominated the market in 2024, favored for its practicality in repeated dosing and outpatient care. The growing adoption of liver-targeted, GalNAc-conjugated therapies is reinforcing this trend.

North America Leads Regional Growth

Why North America? The region is expected to be the fastest-growing market during the forecast period, supported by a dense concentration of RNA drug developers, strong funding environments, and extensive clinical trial activity. A mature ecosystem of CDMOs, analytical laboratories, and regulatory expertise continues to accelerate scale-up and late-stage supply readiness.

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Recent Developments Signal Continued Momentum

March 2025: Alnylam received extended U.S. FDA approval for AMVUTTRA (Vutrisiran) to treat cardiomyopathy associated with transthyretin-mediated amyloidosis (ATTR-CM) in adults, strengthening its cardiovascular outcomes portfolio.

February 2025: Biogen Inc. and Stoke Therapeutics, Inc. announced a collaboration to develop and commercialize Zorevunersen, an investigational antisense oligonucleotide targeting the SCN1A gene for Dravet syndrome.

Why This Market Matters Now

As RNA-based medicines transition from niche innovation to mainstream therapeutic strategy, the oligonucleotide therapeutics market represents a critical growth frontier for pharmaceutical leaders, investors, and strategic partners. Now is the moment to align capabilities, secure supply chain resilience, and invest in next-generation technologies that will define the future of precision medicine.

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