Schedule

Complimentary breakfast will be available outside of Blooming Hill Ballroom.

Speakers: Zollie Yavarow, PhD, Nathan Peck, Conrad "Chris" Weihl, MD, PhD, David Penake, Amy Casey Speaker Bios This opening session sets the stage for the conference by outlining our collaborative goals, providing foundational context on VCP disease and multisystem proteinopathy, and highlighting the patient-centered perspective that drives our research community forward. Attendees will gain insight into the current drug development landscape and understand how this summit aims to advance therapeutic progress through partnership between scientists, clinicians, industry, and the VCP community.

Speakers: Andreas Martin, PhD, Po-lin Chui, PhD, and Sriram Subramaniam, PhD; Chair: Peter Shen, PhD. Associate Professor, University of Utah; Speaker Bios This session examines the structural biology and biochemistry of VCP/p97, including cofactor regulation, cryo-EM structural insights, and computational approaches to understanding protein unfolding mechanisms. Presentation Titles: Andreas Martin, PhD: Modulation of VCP-mediated protein unfolding by Faf1 and other UBX-domain containing cofactors Po-lin Chiu, PhD: Cryo-EM insights into the allosteric regulatory mechanism of p97/VCP ATPase Sriram Subramaniam, PhD: Structural biology and drug discovery in the age of AI

Lunch will be provided with your admission. Offers additional opportunities for networking and scientific exchange.

Speakers: Tarun Kapoor, PhD, Michelle Arkin, PhD, and Marc Diamond, MD; Chair: Michelle Arkin, PhD, Professor and Chair of Pharmaceutical Chemistry, UCSF; Speaker Bios This session examines cofactors and protein-protein interactions of VCP, including chemical and structural probes, VCP-adaptor complex conformations, and the role of VCP in tau prion pathology. Presentation Titles: Tarun Kapoor, PhD: Chemical and structural approaches to examine VCP function Michelle Arkin, PhD: Probing and manipulating VCP conformation in VCP/adaptor complexes Marc Diamond, MD: Amplification and degradation of Tau prions

Speakers: Jordi Diaz-Manera, MD, PhD, Mali Raman, PhD, Firyal Ramzan, PhD; Chair: Tom Lloyd, MD, PhD, Chair of Neurology, Baylor College of Medicine; Speaker Bios This session explores VCP localization and compartment-specific functions, including transcriptomic analysis of muscle pathology, VCP-adaptor roles in ER proteostasis, and lipidation-driven mislocalization in neurodegeneration. Presentation Titles: Jordi Diaz-Manera, MD, PhD: Spatial transcriptomics and single nuclei RNA sequencing reveals compensatory and degenerative pathways in VCP-MSP muscle across histological severity Mali Raman, PhD: The VCP adaptor UBXN1 regulates ATF4-mediated translation restart to maintain ER proteostasis Firyal Ramzan, PhD: Mistargeted by Lipidation: S-acylation and protein mislocalization in neurodegeneration

Join us for an illuminating keynote that traces the scientific journey of discovering ALS as part of the MSP spectrum and explores its implications for VCP disease understanding. Dr. Benatar will share insights from his groundbreaking work in ALS and pre-symptomatic disease research, highlighting how drug development strategies and collaborative research models from the ALS field can inform therapeutic approaches for VCP-MSP. Drawing on his leadership with the CREATE Consortium, Dr. Benatar will discuss lessons learned from building research infrastructure and fostering community collaboration—offering a roadmap for accelerating progress in rare disease research. This keynote sets the foundation for our conference's exploration of translating insights across disease states to advance patient care and treatment development.

Drinks and appetizers provided. Posters available.