Paul Nioi

Company: Alnylam Pharmaceuticals

Job title: Senior Vice President, Research

Seminars:

Panel Discussion: The Future of Oligonucleotide Therapeutics: Where Will the Field Be in 5 Years? 4:00 pm

How will advances in conjugation strategies, nanoparticle formulations, and tissue-specific targeting improve oligonucleotide biodistribution and efficacy? Beyond ALS, Huntington’s Disease & Alzheimer’s. What new indications, such as neurogenetic, epilepsies, neurodevelopmental and neuropsychiatric, are emerging for oligonucleotide-based therapies? What hurdles remain in scaling oligonucleotide production, and how will evolving regulatory frameworks shape the clinical and commercial…Read more

day: Day 2

Reduced ALN-APP for Alzheimer’s Disease & Cerebral Amyloid Angiopathy 8:00 am

Look at work upstream to reduce amyloid production Demonstrate target engagement with the first siRNA to be used in the brain Highlight preclinical findings from AD models and CAA, highlighting reduced cognitive decline in mice and reduced bleeding Delve into clinical data highlighting target engagement and plans for CAARead more

day: Day 1

Panel Discussion: Turning Setbacks into Success: Lessons Learned from Clinical Trial Failures to Strengthen CNS Oligonucleotide Development 9:00 am

Explore common causes of clinical trial failures in CNS oligonucleotide programs, from delivery barriers to patient heterogeneity Share strategies for improving target validation, biomarker selection, and preclinical models to better predict clinical outcomes Discuss best practices for early safety assessment, managing off-target effects, and optimizing dosing strategies Examine how aligning trial design and development plans…Read more

day: Day 1