In the biopharmaceutical arena, the spotlight is increasingly shining on the pioneering field of oligonucleotide therapeutics. The race is on as pharmaceutical titans such as AbbVie, Lilly, Roche, and Takeda display burgeoning interest in licensing innovator oligonucleotides for central nervous system (CNS) indications. This nascent trend is underscored by a staggering 339% surge in total licensing agreement deal value, skyrocketing from £2bn in 2024 to a mind-boggling $2.81bn just a year later.
Leading the charge in this burgeoning field is none other than the US-based Ionis Pharmaceuticals. The company has a robust pipeline of 135 oligonucleotide-based drugs, with a significant portion — 32 to be precise — zeroed in on CNS disorders. Ionis’s bioengineering prowess is already bearing fruit with two of their CNS-focused drugs, Spinraza (nusinersen) for spinal muscular atrophy, and Qalsody (tofersen sodium) for amyotrophic lateral sclerosis, already conquering the market. Both these drugs were developed in tandem with Biogen, as documented in GlobalData’s Pharma Intelligence Center Drugs Database.
But what is sparking this sudden oligonucleotide gold rush? These short synthetic strands of DNA or RNA are the new darlings of precision medicine. They bind selectively to disease-causing genetic sequences, acting as targeted snipers in the battle against complex diseases. The burgeoning interest in oligonucleotide-based therapeutics for CNS disorders is partly due to the expanding application of antisense oligonucleotides (ASOs) and small interfering RNA oligonucleotides (siRNAs).
While ASOs disrupt the production of disease-associated proteins by binding to messenger RNA (mRNA), siRNAs block protein production by triggering the degradation of specific mRNA molecules. This sophisticated genetic ballet is made possible by recent advancements in oligonucleotide synthesis technologies, such as liquid-phase and biocatalytic synthesis methods. These breakthroughs are surmounting long-standing challenges such as scalability, purity, and yield, catapulting oligonucleotides to the forefront of precision medicine for complex conditions, including genetic and neurodegenerative disorders, and cancer.
As per GlobalData’s Pharma Intelligence Center Deals Database, the cumulative total deal value of licensing agreements for innovator oligonucleotides targeting CNS indications reached a whopping $6.05 billion between 2021 and 2025. ASOs accounted for more than half ($3.54 billion), while siRNAs constituted more than a third ($2.51 billion) of these deals. Both modalities have emerged as the go-to platform technologies in the development of oligonucleotide-based drugs for CNS disorders since 2020.
In conclusion, oligonucleotide therapeutics represents one of the most promising frontiers in the biotech landscape. The surge in licensing deals and the interest shown by both pharma giants and leading biotechs like Ionis Pharmaceuticals is an endorsement of the potential that this innovative field offers. As the sector continues to innovate, we can expect further breakthroughs in the treatment of complex diseases and a new era of precision medicine.
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