Licensing agreements for innovator oligonucleotide-based drugs targeting central nervous system (CNS) indications witnessed a dramatic 339% increase in total deal value from 2023 to 2024, reaching $2.81 billion, according to GlobalData's Pharma Intelligence Center Deals Database. This surge reflects renewed industry interest in precision medicine approaches for complex neurological disorders.
Growing Market Momentum
The substantial increase in licensing agreements involving innovator oligonucleotide-based therapeutics for CNS disorders demonstrates the growing application of oligonucleotides, particularly antisense oligonucleotides (ASOs) and antisense RNAi oligonucleotides (siRNAs). These short synthetic strands of DNA or RNA are designed to bind selectively to disease-causing genetic sequences as targeted therapeutics for complex diseases.
ASOs work by binding to messenger RNA (mRNA) to disrupt the production of disease-associated proteins, while siRNAs block protein production by triggering the degradation of specific mRNA molecules. Recent advancements in oligonucleotide synthesis technologies, including liquid-phase and biocatalytic synthesis methods, are accelerating progress by overcoming longstanding challenges with scalability, purity and yield.
Market Composition and Key Players
According to GlobalData's analysis, licensing agreements for innovator oligonucleotides targeting CNS indications secured a cumulative total deal value of $6.05 billion from 2021 to 2025 year-to-date. ASOs accounted for more than half of these deals at $3.54 billion, while siRNAs represented more than a third at $2.51 billion. Both modalities have emerged as the most utilized platform technologies in developing oligonucleotide-based drugs for CNS disorders since 2020.
Large pharmaceutical companies including AbbVie, Lilly, Roche and Takeda have demonstrated strong interest in licensing innovator oligonucleotides for CNS indications, accounting for 63% of licensing deals between 2021 and 2025 year-to-date.
Industry Leadership
US-headquartered biotech Ionis Pharmaceuticals remains at the forefront of the growing oligonucleotide therapeutics field, having out-licensed assets for a total of $13.4 billion over the past decade. The company currently maintains 135 oligonucleotide-based drugs in its pipeline, with 32 targeting CNS disorders. Two of these have reached the market: Spinraza (nusinersen) for spinal muscular atrophy and Qalsody (tofersen sodium), co-developed and licensed by Biogen for amyotrophic lateral sclerosis.
Record-Breaking 2025 Deal
Arrowhead Pharmaceuticals secured the largest deal of 2025 through an exclusive global licensing and collaborative agreement with Sarepta Therapeutics. This agreement, valued at up to $2.18 billion, grants Sarepta rights to multiple programs across clinical, preclinical and discovery stages, focused on rare, genetic and CNS diseases.
Market Outlook
Oligonucleotides are becoming an increasingly attractive focus for biopharmaceutical licensing, driven by advances in manufacturing and expanding therapeutic potential across a range of conditions, as well as the relative efficiency of their synthesis compared to small molecules or biologics. These developments are paving the way for more efficient large-scale production and reinforcing the role of oligonucleotides as a cornerstone of precision medicine for complex conditions such as genetic and neurodegenerative disorders, and cancer.
With momentum building around these advanced modalities, the oligonucleotide market is well-positioned to drive the next wave of personalized treatment in the coming years.
