Perfect Your Oligonucleotide Pipeline to Slow, Cure & Prevent Neurodegeneration
In a landscape where traditional treatments often fall short, oligonucleotide therapeutics have emerged as game-changers delivering precise targeting and potentially life-altering outcomes in neuroscience. As neurology overtakes cancer as the number one indication prioritized by oligonucleotide drug developers, now is the time to stay at the forefront of ASO and siRNA innovation.
The 5th Oligonucleotides for CNS Summit offered you the chance to be a part of critical conversations discussing the latest brain shuttle innovation with Regeneron and Eli Lilly, translational tools including BBBoids, cryptic exon inclusion sites, derisking nonclinical toxicology with Servier and Roche, PKPD modelling and dose selection, ProQR's RNA editing, Novo Nordisk's neurometabolic modulation, Alnylam Pharmaceuticals' biomimetic chemistry and more.
What Was New for 2025?
Unpublished insights and never-seen-before data
Two tracked workshop day designed for chemists and biologists
10 new presenting companies including Denali Therapeutics, Alnylam Pharmaceuticals and Novo Nordisk
Regulator presence from Ron Wange, a former FDA expert and author of the non-clinical guidance for oligonucleotides
A greater spread of content across neurogenetic, neurodegeneration, neuromuscular, neurodevelopmental, rare, and ultra-rare indications, including Angelman’s syndrome, epilepsies and more!
Delegates Joined Neurobiologists & Oligonucleotide Chemists to:
Discover novel conjugates beyond antibodies and TfR1, and optimize oligonucleotide chemistry, to enhance potency, safety and stability
Advance more efficient delivery systems to ensure target-specific CNS tropism with minimum efficacious doses
Explore the latest in vitro and in vivo models to test new compounds and optimize CNS biomarkers to assess oligonucleotide efficacy
Review innovative strategies for assessing ASO and siRNA delivery and distribution in the CNS
Uncover the latest novel approaches, including RNA editing and splice modulation, and foster cross-modality learnings
Who Did You Meet?
The 5th Oligonucleotides for CNS meeting united 100+ key pharma and biotech across discovery biology, oligonucleotide chemistry, DMPK, pharmacology, CMC and platform technology.
Delegates connected with C-Level executives to senior scientists, to hear the latest unpublished data, participate in heated panel discussions, attended structured group discussions and took a deep dive into workshop topics, uniquely available to this meeting.
Peer Perspectives:
"I was able to interact with experts in the field who were encountering the same challenges in ASO drug development. The intimate setting enabled easy flow of communication and open discussion between participants"
Director, Clinical Pharmacology, Servier, Past Attendee
"It was great to meet other people in the field and get their perspectives on different modalities"
Quantitative Systems Pharmacology Modeler Professional, Takeda, Past Attendee