The Rare Disease Desert Symposium will officially kick off at 1pm MST on Monday, February 26th, with registration at Icagen-T in Tucson, AZ. Attendees coming from the east or west coast of the US will be able to travel that morning, as there are several early flight options to both Tucson and Phoenix. Alternatively, attendees can arrive Sunday, February 25th, and spend Monday morning at Icagen-T for a guided site tour.
Please note the following agenda is in Mountain Standard Time (MST).
Day 1: Monday, February 26, 2018
|2:00pm||Welcome and Opening Remarks
Ken Wertman, Ph.D., SVP and Site Director, Icagen
Prof. Louis Kunkel
"Duchenne Muscular Dystrophy: 30 years post dystrophin"
Muscular Dystrophies 1: DMD
Paul August, Ph.D., VP, Biology, Icagen (Chair)
|3:00pm||Laurie Conklin, M.D.
Director, Clinical Programs, ReveraGen Biopharma, and Assistant Professor of Pediatrics at George Washington University School of Medicine
"Context of use and fit for purpose: Integration of pharmacodynamic biomarkers into the vamorolone clinical program"
|3:30pm||Coffee & Networking Break|
|4:00pm||Prof. Andy Berglund
Department of Biochemistry and Molecular Biology, Center for NeuroGenetics, University of Florida
"Exploiting a chink in the armor of myotonic dystrophy to develop novel therapeutic compounds"
|4:30pm||Prof. Rita Perlingeiro
Cardiovascular Division, University of Minnesota; Lillehei Professor in Stem Cell and Regenerative Cardiovascular Medicine, Lillehei Heart Institute
"Pluripotent Stem Cells in DMD: Potential for Cell Therapy, Disease Modelling, and Drug Discovery"
|5:00pm||Dr. Sue Shackleton
Dept. of Molecular and Cell Biology, University of Leicester, UK
"The missing LINC between the nuclear envelope and rare myopathies"
|5:30pm||Dr. Jane Larkindale
Executive Director, Duchenne Regulatory Science Consortium (D-RSC), Critical Path Institute
"Data Sharing and Development of Drug Development Tools through the Duchenne Regulatory Science Consortium (D-RSC) to Accelerate Drug Development"
Doug Krafte, Ph.D., Chief Scientific Officer, Icagen
Hilton Tucson El Conquistador
Day 2: Tuesday, February 27, 2018
|8:00am||Continental Breakfast & Registration|
Emily Kamer-Golinkoff, Co-Founder of Emily's Entourage
Dr. Richard Gregory, Chief Scientific Officer, ImmunoGen
Rare Disease – From Monogenic Diseases to Cancer"
Muscular Dystrophies 2: FSHD & EDMD
Prof. Michael Kyba (Chair)
|9:15am||Angela Cacace, Ph.D.
Vice President, Biology, Fulcrum Therapeutics
"Balancing Gene Regulation in an Era of Precision Medicine: FXS and FSHD"
|9:45am||Prof. Michael Kyba
Lillehei Endowed Scholar, Carrie Ramey / CCRF Endowed Professor in Pediatric Cancer Research, Department of Pediatrics
"FSHD: Disease models and prospects for therapy"
|10:15am||Peter L. Jones, PhD
Mick Hitchcock, PhD Endowed Chair in Medical Biochemistry, Associate Professor of Pharmacology, Center for Molecular Medicine, University of Nevada, Reno School of Medicine
"Targeting FSHD epigenetics for diagnostics and therapy"
|10:45am||Coffee & Networking Break|
Other Rare Diseases
|11:00am||Dr. Brady Maher
Lead Investigator at the Lieber Institute for Brain Development, Johns Hopkins University
"Identification and validation of SCN10a/Nav1.8 as a therapeutic target for Pitt Hopkins Syndrome"
Dr. Robert Ring
|12:00am||Dr. Christopher VanDeusen
President and co-Founder, Omdana Therapeutics
"Understanding the Relaxin Pathway and its impact on Pulmonary Hypertension"
|12:30pm||Lunch Break & Posters|
Prof. Justin Ichida (Chair)
|2:00pm||Prof. Justin Ichida
Stem Cell & Regenerative Medicine, Keck School of Medicine, University of Southern California
"Identification of Therapeutic Targets for ALS and FTD Using Phenotypic Screens on Patient-derived Models"
|2:30pm||Rita Sattler, PhD.
Associate Professor of Neurobiology and Neurology, Barrow Neurological Institute, Dignity Health, St. Joseph's Hospital and Medical Center
"Patient-derived induced pluripotent stem cells to study mechanisms of disease in ALS/FTD"
|3:00pm||Evangelos Kiskinis, PhD.
Assistant Professor of Neurology & Physiology
Director Stem Cell Core Facility
Northwestern University Feinberg School of Medicine
"Using iPSCs to Study the Development and Function of Human Motor Neurons in Health and Disease"
Martin Mense, Ph.D. (Chair)
|4:00pm||Prof. Robert Bridges
Physiology and Biophysics, Chicago Medical School
"Advancing the search for epithelial ion channel modulators"
|4:30pm||Dr. Martin Mense
Vice President for Drug Discovery, Cystic Fibrosis Foundation Therapeutics
"Beyond CFTR F508del – Research at the CFFT Lab"
|5:00pm||Dr. Tim Young
Research Fellow at Vertex
"Developing medicines for all people with CF"
|5:30pm||Close of Symposium and Closing Remarks
Richie Cunningham, CEO, Icagen
|6:00pm||Reception on deck at Icagen-T for those remaining|