Brian Blagg, Paul Helquist, Olaf Wiest & Warren Family Research Center for Drug Discovery and Development, University of Notre Dame - Title: Design and Synthesis of Bio-organic Small Molecule Tools for Investigations of Niemann-Pick Type C Disease

Ben Calverley, The Scripps Research Institute - Title: Utilizing Artificial Intelligence to Mine Genetic Variation in the NPC1 Population for Corrective Therapeutics

Stephanie Cologna, Associate Professor, University of Illinois Chicago & Denny Porter, senior Investigator, National Institutes of Health; Title - Plasma Protein Biomarkers of NPC1

Thomas Grewal, Associate Professor, University of Sydney; Title:  Targeting Rab7 and TBC1D15 to normalize cellular cholesterol distribution in NPC1 disease

Daniel Heller, Assistant Member, Memorial Sloan Kettering Cancer Center; Title - Targeting Autophagic-Lysosomal Pathway Dysregulation to Improve Treatment of Niemann-Pick Type C Disease

Mike Henne, Assistant Professor, UT Southwestern – Title:  Dissecting the role of SNX-RGS proteins in inter-organelle cholesterol metabolism 

Marc Patterson and Conan Donnelly, International Niemann-Pick Disease Registry - Title: Growing the patient-led International Niemann-Pick Disease Registry in the United States & Canada

Fred Maxfield, Professor & Chairman of Biochemistry and Cell & Developmental Biology, Cornell - Title: Systematic examination of the role of chaperones in folding the NPC1 protein

Marc Patterson and Conan Donnelly, International Niemann-Pick Disease Registry - Title: Accelerating Progress by Facilitating Registry Recruitment in the USA

Suzanne Pfeffer, Professor of Biochemistry, Stanford University - Title: Modifiers of NPC1-mediated cholesterol export from lysosomes

John Pluvinage, Resident Physician of Neurology UCSF & Charles Vite, Professsor of Neurology, University of Pennsylvania; Title: Preclinical evaluation of a therapeutic CD22 antibody in feline Niemann-Pick Type C1 disease

Denny Porter, senior Investigator, National Institutes of Health; Title:  Investigation of mechanism of action and in vivo translation of candidate therapeutic compounds identified in neuron-based high-throughput drug and CRISPRi screens

Jorge L. Rodriguez-Gil, Resident Physician, Stanford & Jacquelin Butterfield; Research Associate, Stanford; Title: Transcriptomics as a tool for biomarker and drug discovery in Nieman-Pick Disease type C1

Rajat Rohatgi, Associate Professor, Stanford & Maia Kinnebrew, Postdoctoral Fellow, Stanford; Lipid trafficking pathways in Niemann-Pick Type C disease

Marco Segatto, Associate Professor of Physiology, University of Molise; Title:  p75NTR targeting as a novel approach to combat Niemann-Pick type C1 disease

Judith Storch, Professor, Rutgers - Title: Translational Potential of LBPA for NPC disease

Charles Vite, Professor, University of Pennsylvania - Support of the NPC1 cat colony for future preclinical trials

Hongyuan Yang, Professor, University New South Wales - Title: The role of phosphatidylserine, ORPs and GRAMD1s in lysosomal cholesterol trafficking

Melissa Wasserstein, MD, Einstein School of Medicine - Title: New Born Screen (Screen Plus)

Roberto Zoncu, Associate Professor, and Paulina Ordonez, Assistant Professor, University of California, San Diego - Title: Decoding cellular dysfunction in Niemann-Pick type C through functional genomics and organelle metabolomics

Biomarker Initiative: Biomarker analysis with Omicera Diagnostics & collaboration with the National Institutes of Health and Rush Hospital;  Title: Analysis of Patient Samples to Identify Candidate Biomarkers