Sunday
2:00 pm - 9:00 pm
Arrival and Check-in
6:00 pm - 7:00 pm
Dinner
7:30 pm - 7:40 pm
Introductory Comments by GRC Staff / Welcome and Introduction from the Chairs
7:40 pm - 9:30 pm
Keynote Session: Translational Science and Therapeutic Landscape
Discussion Leaders: Charlotte Sumner (Johns Hopkins University School of Medicine, United States)
7:40 pm - 8:00 pm
Introduction by Discussion Leader
8:00 pm - 8:45 pm
Don Cleveland (Ludwig Institute for Cancer Research, United States)
"Regulated Multi-Phase Transitions: TDP-43, Anisosomes, Aggregation, and Neurodegeneration"
8:45 pm - 9:10 pm
Discussion
9:10 pm - 9:30 pm
General Discussion
Monday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
TDP-43 and Neurodegeneration: Loss of Function, Cryptic Splicing, and Therapeutics
Discussion Leader: Emanuele Buratti (ICGEB, Italy)
9:00 am - 9:15 am
Introduction by Discussion Leader
9:15 am - 9:35 am
Pietro Fratta (University College London, United Kingdom)
"When, Where and How to Stop TDP-43 Cryptic Splicing"
9:35 am - 9:45 am
Discussion
9:45 am - 10:05 am
Clotilde Lagier-Tourenne (Harvard Medical School / Massachusetts General Hospital, United States)
"Stathmin-2: An Emerging Therapeutic Target in TDP-43 Proteinopathies"
10:05 am - 10:15 am
Discussion
10:15 am - 10:40 am
Coffee Break
10:40 am - 11:00 am
Magdalini Polymenidou (University of Zurich, Switzerland)
"RNA Binding Proteins Aggregation in ALS"
11:00 am - 11:10 am
Discussion
11:10 am - 11:30 am
Yuna Ayala (Saint Louis University, United States)
"Binding to RNA and Protein Assembly Regulate TDP-43 Homeostasis and Cellular Dynamics"
11:30 am - 11:40 am
Discussion
11:40 am - 12:00 pm
Leonard Petrucelli (Mayo Clinic College of Medicine and Science, United States)
"TDP-43 Splicing Biology and Cryptic Peptides as Potential Biomarkers"
12:00 pm - 12:10 pm
Discussion
12:10 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Group Photo / Lunch
1:30 pm - 4:30 pm
Free Time
3:00 pm - 4:00 pm
The GRC Power Hourâ„¢
The GRC Power Hourâ„¢ is designed to address diversity and inclusion in the scientific workplace by providing a safe environment for informal and meaningful conversations amongst colleagues of all career stages. The program supports the professional growth of all members of our communities, including ethnicity, race and/or gender identity by providing an open forum for discussion and mentoring.
Organizer: Aarti Sharma (Regeneron, United States)
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Emerging Genetic and Mechanistic Modifiers of ALS and FTD
Discussion Leader: Rosa Rademakers (University of Antwerp & Flemish Institute for Biotechnology (VIB), Belgium)
6:00 pm - 6:20 pm
Introduction by Discussion Leader
6:20 pm - 6:40 pm
Benjamin Ryskeldi-Falcon (MRC Laboratory of Molecular Biology, United Kingdom)
"Structures of Pathological TDP-43 Filaments in ALS and FTLD"
6:40 pm - 6:50 pm
Discussion
6:50 pm - 7:10 pm
Dorothee Dormann (Johannes Gutenberg University (JGU) Mainz, Germany)
"Molecular Mechanisms of RNA Binding Protein Dysfunction in ALS and Related Neurodegenerative Disorders"
7:10 pm - 7:20 pm
Discussion
7:20 pm - 7:40 pm
Sami Barmada (University of Michigan, United States)
"TDP43 Splice Variants: Friend or Foe?"
7:40 pm - 7:50 pm
Discussion
7:50 pm - 8:00 pm
General Discussion
8:00 pm - 9:00 pm
Dinner
Tuesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Axonal and Synapse Biology; Degeneration in Motor Neuron Diseases
Discussion Leader: Jeroen Pasterkamp (University Medical Center Utrecht, The Netherlands)
9:00 am - 9:20 am
Introduction by Discussion Leader
9:20 am - 9:40 am
Alison Twelvetrees (The University of Sheffield, United Kingdom)
"Motor Proteins in Motor Neuron Diseases; Can We Get There in Time?"
9:40 am - 9:50 am
Discussion
9:50 am - 10:10 am
Robert Burgess (The Jackson Laboratory, United States)
"tRNA Synthetase-Associated Peripheral Neuropathies: Mechanisms and Therapeutic Strategies"
10:10 am - 10:20 am
Discussion
10:20 am - 10:50 am
Coffee Break
10:50 am - 11:10 am
Eran Perlson (Tel Aviv University, Israel)
"Is ALS a Distal Disease? The Interplay Between Local Synthesis and Neuromuscular Degeneration"
11:10 am - 11:20 am
Discussion
11:20 am - 11:40 am
Sandrine Da Cruz (VIB- KU Leuven Center for Brain and Disease Research, Belgium)
"Uncovering Mechanisms of Axon Maintenance and Demise in ALS"
11:40 am - 11:50 am
Discussion
11:50 am - 12:10 pm
Short Talk Selected from Poster Abstracts
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Neuroinflammation and Vascular Biology in ALS and FTD
Discussion Leader: Severine Boillee (ICM - INSERM - Sorbonne University, France)
6:00 pm - 6:20 pm
Introduction by Discussion Leader
6:20 pm - 6:40 pm
Eric Huang (University of California, San Francisco, United States)
"Neuroimmune Dysfunction in FTD-ALS Spectrum Disorders"
6:40 pm - 6:50 pm
Discussion
6:50 pm - 7:10 pm
Renzo Mancuso (VIB-Center for Molecular Neurology and University of Antwerp, Belgium)
"Impact of C9orf72 Mutations in the Biology of Microglia"
7:10 pm - 7:20 pm
Discussion
7:20 pm - 7:45 pm
Late-Breaking Topic
7:45 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Wednesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Astrocyte Biology in ALS and FTD
Discussion Leader: Jeffrey Rothstein (Johns Hopkins University, United States)
9:00 am - 9:15 am
Introduction by Discussion Leader
9:15 am - 9:35 am
Brigitte Van Zundert (Andres Bello University, Chile)
"A New Role for an Archaic Model: Release of Inorganic Polyphosphate byALS/FTD Astrocytes Causes Non-Cell-Autonomous Toxicity to Motoneurons"
9:35 am - 9:45 am
Discussion
9:45 am - 10:05 am
Kristine Freude (University Copenhagen, Denmark)
"The Involvement of Glia in Frontotemporal Dementia"
10:05 am - 10:15 am
Discussion
10:15 am - 10:45 am
Coffee Break
10:45 am - 11:05 am
Shane Liddelow (NYU Grossman School of Medicine, United States)
"Glial Signaling and Neurodegeneration"
11:05 am - 11:15 am
Discussion
11:15 am - 11:25 am
Short Talk Selected from Poster Abstracts
11:25 am - 11:30 am
Discussion
11:30 am - 11:50 am
Clive Svendsen (Cedars-Sinai Medical Center, United States)
"Astrocytes for Neuroprotection"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:20 pm
Alyssa Coyne (Johns Hopkins University School of Medicine, United States)
"Aberrant Activation of CHMP7/ESCRT-III Nuclear Pore Complex Surveillance as an Initiator of Pathophysiological Cascades in ALS/FTD"
12:20 pm - 12:30 pm
Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Translational Biomarkers
Discussion Leader: Lucie Bruijn (Novartis, Switzerland)
6:00 pm - 6:20 pm
Introduction by Discussion Leader
6:20 pm - 6:40 pm
David Owen (Imperial College London, United Kingdom)
"Species Differences in Interpretation of the TSPO PET Signal"
6:40 pm - 6:45 pm
Discussion
6:45 pm - 7:05 pm
Sanjay Kalra (University of Alberta, Canada)
"Progressive Motor and Extra-Motor Cerebral Degeneration in ALS"
7:05 pm - 7:10 pm
Discussion
7:10 pm - 7:30 pm
Andrea Malaspina (QS MND CENTRE, INSTITUTE OF NEUROLOGY, UCL, United Kingdom)
"Emerging Pathological Mechanism and Biomarkers in Amyotrophic Lateral Sclerosis"
7:30 pm - 7:35 pm
Discussion
7:35 pm - 7:50 pm
Short Talk Selected from Poster Abstracts
7:50 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Thursday
7:30 am - 8:30 am
Breakfast
8:30 am - 9:00 am
Business Meeting
Nominations for the Next Vice Chair(s); Complete the GRC Evaluation Forms; Discuss Future Dates and Venue; Election of the Next Vice Chair(s)
9:00 am - 12:30 pm
Translational Science in ALS and FTD
Discussion Leaders: David Taylor (ALS Society of Canada, Canada)
9:00 am - 9:10 am
Introduction by Discussion Leader
9:10 am - 9:30 am
Timothy Miller (Washington University in St. Louis, United States)
"RNA-Targeted Therapies for ALS"
9:30 am - 9:40 am
Discussion
9:40 am - 10:00 am
Neil Shneider (Columbia University, United States)
"Silencing FUS and Other Rare Genetic Forms of ALS"
10:00 am - 10:10 am
Discussion
10:10 am - 10:40 am
Coffee Break
10:40 am - 11:00 am
Paymaan Jafar-nejad (Ionis Pharmaceuticals, United States)
"Antisense Oligonucleotides (ASOs) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)"
11:00 am - 11:10 am
Discussion
11:10 am - 11:30 am
Steven Gray (University of Texas Southwestern Medical Center, United States)
"Standardized Translational Approach to Target Motor and Sensory Neurons Via Intrathecal Administration of AAV9"
11:30 am - 11:40 am
Discussion
11:40 am - 12:00 pm
John Day (Stanford University, United States)
"Lessons Learned from Treating Spinal Muscular Atrophy"
12:00 pm - 12:10 pm
Discussion
12:10 pm - 12:25 pm
Short Talk Selected from Poster Abstracts
12:25 pm - 12:30 pm
Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Late-Breaking Topics
Discussion Leaders: Don Cleveland (Ludwig Institute for Cancer Research, United States)
6:00 pm - 6:20 pm
Michael Ward (National Institutes of Health, United States)
"TDP-43 in ALS"
6:20 pm - 6:30 pm
Discussion
6:30 pm - 6:50 pm
Chris Henderson (Biogen, United States)
"Translatable Endpoints and Biomarkers to Increase Probability of Success in ALS Drug Development"
6:50 pm - 7:00 pm
Discussion
7:00 pm - 7:20 pm
Christopher Pearson (The Hospital for Sick Children, Canada)
"New Epigenetic Awareness at an ALS Locus"
7:20 pm - 7:30 pm
Discussion
7:30 pm - 7:50 pm
Aaron Gitler (Stanford University, United States)
"Two New ALS Drug Targets (And One Drug) "
7:50 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Friday
7:30 am - 8:30 am
Breakfast
9:00 am
Departure