From Discovery to Deals: The Neurological Oligonucleotide Landscape at a Glance

Presented at the 5th Oligonucleotides for CNS Summit in August 2025, this Beacon RNA analysis spotlights the latest regulatory milestones, target pipelines, and deal-making shaping oligonucleotide drug development. Gain a detailed view into the companies, trials, and technologies driving the future of neurological therapies.

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Why This Neurological Oligonucleotide Data Is Essential

This presentation is your competitive advantage. Here’s what you’ll gain access to:

Preclinical, Clinical & Regulatory Insights

  • Of 2,270 oligonucleotide therapies in development, 44% target neurological disorders, highlighting the central role CNS plays in this modality.
  • The neurological pipeline is led by targets such as DMD, HTT, and SOD1, alongside other high-interest genes driving R&D focus.
  • Regulatory momentum is building: Dyne Therapeutics’ DYNE-101 has received Breakthrough Therapy and Fast Track Designation for MDT1, setting a precedent for future oligonucleotide approvals.

Oligonucleotide Deal-Making & Collaborations

  • Oligonucleotide partnerships continue to concentrate on neurology, with 27 collaborations focused on CNS disorders.
  • Deal-making underscores investor and developer confidence in the therapeutic potential of oligonucleotides for neurological disease.

Company & Pipeline Landscape

  • More than 560 active biopharma companies in the RNA space have at least one RNA-based therapeutic in preclinical development.
  • While most are focused on mRNA and RNAi, oligonucleotide developers are rapidly advancing their presence in neurology.

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