SUNDAY, MARCH 25 | 5:00 - 8:00 PM
SC1: Oligonucleotide Therapeutics: From Discovery to Manufacturing
RNAi/antisense oligonucleotides can target virtually any disease-causing gene and promise to become a third major class of therapeutics (besides small molecules and biologics). For this to happen, however, several challenges have to be addressed. This course discusses considerations taken into account when selecting oligonucleotide therapeutic programs: from target and delivery selection to developmental and manufacturing particularities. Detailed discussion will focus on, but not be limited to, the tightly interconnected factors of therapeutic indication, delivery and targets, as well as chemistry manufacturing and controls (CMC), regulatory, cost, and intellectual property considerations. Developers who are seeking a comprehensive and up-to-date overview from recognized oligonucleotide development experts are highly encouraged to join.
Dmitry Samarsky, PhD, CSO, Silence Therapeutics
Muthiah (Mano) Manoharan, PhD, Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc.
Thazha P. Prakash, Director, Medicinal Chemistry, Ionis Pharmaceuticals
Konstantin M. Linnik, PhD, Partner, Intellectual Property, Nutter, McClennen & Fish, LLP; former Lead Patent Counsel for Oligonucleotide Therapeutics, Pfizer, Inc.
MONDAY, MARCH 26 | 6:30 - 9:30 PM
SC2: Overcoming Challenges with Peptide Delivery
Peptides make attractive drug candidates due to their specificity, potency and low toxicity, but present particular challenges for their delivery to the site of action, due to their short half-life and susceptibility to proteolytic degradation. This short course reviews the latest challenges in peptide drug delivery and the various options available, including oral, transdermal and nanosystems. We also look at challenges around half-life, bioavailability, stability and formulation.
Joël Richard, PhD, Senior Vice President, Peptides Development, Ipsen
TUESDAY, MARCH 27 | 5:30 - 8:30 PM
SC3: CRISPR-Based Gene Editing for Targeted Therapies
While the challenges and risks associated with oligonucleotide therapies still remain, there is a new and better understanding of how genes can be effectively manipulated and delivered. With the rise of gene editing tools and enhanced knowledge of targeted delivery, these therapeutic modalities are once again being embraced with renewed hope and enthusiasm. This course helps you understand how gene editing – particularly the one enabled by the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas9 system – works, and how it can be used to help develop targeted therapies with good efficacy and delivery.
Clifford Steer, MD, Professor of Medicine and Genetics, Cell Biology, and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School
Ciro Bonetti, PhD, Scientist, Regeneron Pharmaceuticals
Eric B. Kmiec, PhD, Director, Gene Editing Institute; Senior Research Scientist, Center for Translational Cancer Research, Helen F. Graham Cancer Center & Research Institute, Christiana Care Health System
* Separate registration required.