Muthiah Manoharan

Company: Alnylam Pharmaceuticals

Job title: SVP

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

Biomimetic Chemistry of siRNA Delivery to CNS 3:00 pm

Synthetic small interfering RNAs (siRNAs) are potent inhibitors of gene expression. These molecules are perfect examples of biomimetic chemistry as synthetic siRNAs act through the natural RNA interference (RNAi) pathway To deliver therapeutic siRNAs for CNS applications, we have developed approaches that include extensive chemical modification of the siRNAs and lipidic nucleic acids (siRNA-lipid conjugates)…Read more

day: Day 1