2017 Short Courses*

SUNDAY, MARCH 26 | 5:00 - 8:00 PM
Pre-Conference Dinner Short Courses

SC1: Oligonucleotide Therapeutics: From Discovery to Manufacturing


Dmitry Samarsky, Ph.D., CSO, Silence Therapeutics

Muthiah (Mano) Manoharan, Ph.D., Senior Vice President, Drug Discovery, Alnylam Pharmaceuticals, Inc.

Thazha P. Prakash, Director, Medicinal Chemistry, Ionis Pharmaceuticals

Steffen Schubert, Ph.D., Head, In Vitro Pharmacology – Drug Discovery, Silence Therapeutics

Konstantin M. Linnik, Ph.D., Partner, Intellectual Property, Nutter, McClennen & Fish, LLP; former Lead Patent Counsel for Oligonucleotide Therapeutics at Pfizer, Inc. 

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.   

MONDAY, MARCH 27 | 6:30 - 9:30 PM
Dinner Short Courses

SC3: CRISPR-Based Gene Editing for Targeted Therapies - Detailed Agenda

Instructors: Clifford Steer, M.D., Professor of Medicine and Genetics, Cell Biology, and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School

Ciro Bonetti, Ph.D., Scientist, Regeneron Pharmaceuticals

Hao Yin, Ph.D., Research Scientist, The David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

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.

* Separate registration required.