Program

Optimization of RNase H-Dependent Antisense Drugs for the Treatment of Myotonic Dystrophy (C. Frank Bennett, Ph.D., senior vice president of research, Ionis Pharmaceuticals)

This session covers all aspects of the mutations associated with DM, including population genetics, particularities of the mutations, epigenetic modifications and repeat instability mechanisms.

This session covers the research devoted to understanding DM disease mechanisms in various model systems, with the exception of repeat instability mechanisms covered in Session 1.

This session covers preclinical and clinical studies of the underlying mechanisms and progression of cancer and premature aging in DM.

This session covers all clinical aspects of DM and its burden on patients and caregivers, as well as ethical and social issues in DM patients and families.

This session covers organ-specific phenotypes of DM and practices for their management and treatment.

This session covers in vitro model systems, their development and use in mechanistic studies or therapy development.

This session covers in vivo model systems, their development and use in mechanistic studies or therapy development.

Lessons from DMPK-Rx clinical trials in myotonic dystrophy (Laurence Mignon, Ph.D.)

This session covers clinical trial readiness resources and tools used in the clinical development of new therapeutic strategies.

Note: There is a bridge walkway directly from the Hyatt Regency San Francisco to the Sens Restaurant in the Embarcadero Center. Attire: Business Casual.

The annual Drug Development Roundtable brings together leaders from academia, biotechnology and pharmaceutical industries to discuss opportunities, barriers and pre-competitive space collaborations to facilitate the pathway to drugs and biologics for DM.

In response to requests from drug development professionals to help identify CNS endpoints appropriate for the regulatory review process, and to accelerate the development of CNS-targeting DM therapies, IDMC-11 and the Myotonic Dystrophy Foundation will jointly host a multi-panel discussion to bring patient perspectives on disease burden and clinically meaningful endpoints to efforts to develop endpoints for CNS-targeting DM therapies. The meeting format will mirror the very successful Patient-Focused Drug Development (PFDD) meeting hosted by MDF with the FDA in September 2016. Panels will be seated with people affected by DM, and DM caregivers. The session will be moderated by James Valentine, J.D., who also moderated the September 2016 PFDD meeting. The session will begin with a presentation on outcome measure development to help the lay audience understand the importance and role of endpoints in drug development and approval, and to frame the goals for the session. Panel presentations will be followed by moderated audience discussion and a presentation from an industry representative regarding possible future DM CNS endpoints, and CNS endpoints used in other neurological diseases that might inform upcoming drug development efforts in DM. A publication summarizing the workshop is planned.

Representatives from companies and academia will provide updates on early stage and advanced drug development efforts in the DM field.