CNS innovator oligonucleotide licensing value surges by 339% to $2.8 billion in 2024, reveals GlobalData

New Delhi: Innovator oligonucleotide-based drugs targeting central nervous system (CNS) disorders have seen a remarkable 339% increase in licensing agreement deal value, rising from $640 million in 2023 to $2.81 billion in 2024, according to GlobalData, a leading data and analytics company.

Oligonucleotides, which are short synthetic DNA or RNA strands, are utilized in precision medicine to selectively bind to disease-causing genetic sequences, offering targeted therapeutic options for complex diseases.

Ophelia Chan, Senior Business Fundamentals Analyst at GlobalData, comments: “The surge in licensing agreements is largely attributed to the growing application of antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs). ASOs disrupt the production of disease-associated proteins by binding to messenger RNA (mRNA), while siRNAs inhibit protein production by degrading specific mRNA molecules.

“Recent advancements in oligonucleotide synthesis technologies, including liquid-phase and biocatalytic methods, have addressed challenges related to scalability, purity, and yield, facilitating more efficient large-scale production. These innovations reinforce the role of oligonucleotides in precision medicine, particularly for genetic, neurodegenerative disorders, and cancer.”

According to GlobalData’s Pharmaceutical Intelligence Center Deals Database, licensing agreements for innovator oligonucleotides targeting CNS indications secured a cumulative total deal value of $6.05 billion from 2021 to 2025 YTD, with ASOs contributing over half ($3.54 billion) and siRNAs accounting for more than a third ($2.51 billion).

Chan adds: “Both modalities have emerged as the most used platform technologies in developing oligonucleotide-based drugs for CNS disorders over the past five years.”

Large pharmaceutical companies like AbbVie, Eli Lilly, Roche, and Takeda have shown significant interest, representing 63% of licensing deals from 2021 to 2025 YTD. However, Ionis Pharmaceuticals, leads the field with $13.4 billion in out-licensed assets over the past decade and a pipeline of 135 oligonucleotide-based drugs, including two approved treatments for CNS disorders: Spinraza (nusinersen) for spinal muscular atrophy and Qalsody (tofersen sodium), co-developed and licensed by Biogen for amyotrophic lateral sclerosis.

Additionally, Arrowhead Pharmaceuticals made headlines in 2024 with a $2.18 billion licensing agreement with Sarepta Therapeutics, covering multiple programs for rare genetic and CNS diseases – the largest deal of the year.

Chan concludes: “The increasing focus on oligonucleotides in biopharmaceutical licensing is driven by advancements in manufacturing and their therapeutic potential, positioning the oligonucleotide market to spearhead the next wave of personalized treatments in the coming years.”

Note: A single deal may include multiple drugs across various indications and modalities. This figure includes all announced and completed innovator CNS oligonucleotide licensing agreements across all active development stages (marketed, pre-registration, Phase III, Phase II, Phase I, pre-clinical, and discovery) globally from 2021 to 2025 YTD with a reported deal value.