ORS Musculoskeletal Biology Workshop at Sun Valley Agenda

7:00 AM – 8:00 AM
Breakfast

7:30 AM – 8:00 AM
Ultragenyx Breakfast Session
Understanding Bone Remodeling in XLH:  Insight into Restoring Phosphate Homeostasis
Presenter: Javier San Martin. MD

8:00 AM – 9:00 AM
ASBMR/Harold M. Frost Young Investigator Awards Presentations
Chair: Alexander Robling, PhD, Indiana University

9:15 AM – 12:00 PM
Session: Epigenetics and Musculoskeletal Disease
Chair:  Regis J. O’Keefe, MD, PhD, Washington University

Brief Overview of Epigenetics
Regis J. O’Keefe, MD, PhD, Washington University

Histone Deacetylase 3 in Cartilage Development and Osteoarthritis
Jennifer J. Westendorf, PhD, Mayo Clinic:

Defining DNMT3b and Downstream Targets in the Pathogenesis of Osteoarthritis
Regis J. O’Keefe, MD, PhD, Washington University

Genome-wide DNA Methylation Changes in Mice Under Simulated Microgravity Conditions
Mario Fraga, University of Oviedo

1:00 PM – 2:30 PM
Career Development Workshop: Bringing Discoveries to Market: Navigating the FDA
Chair: Jose Moreno, PhD, FDA

The workshop will provide the audience with an overview of the various mechanisms available to obtain FDA feedback regarding the potential regulatory pathway ahead for any products resulting from their research efforts. This information is of special interest for researches applying to the Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) funding programs, where such regulatory information can have a larger impact in the funding process.  Attendees will gain key knowledge regarding the current regulatory pathways for device, drug, biological and combination products, and the mechanisms available to present the results of their research efforts to FDA.

7:30 PM – 9:30 PM
Session: Rare Diseases of Phosphate Handling and Tissue Mineralization: Too Much or Too Little
Chair:  Javier San Martin, MD, Ultragenyx, Inc.

Introduction to Phosphate Metabolism
Javier San Martin, MD, Ultragenyx, Inc.

From Therapeutics Target Identification to Drug Development, with an Anti-FGF23 Antibody, Burosumab
Javier San Martin, Ultragenyx, Inc.

Targeting a Unique Pathophysiology for Improved Outcomes in X-linked Hypophosphatemia
Thomas Carpenter; MD, Yale University

Multiple Osteochondroma and Fibrodysplasia Ossificans Progressiva: Two Distinct Pediatric Disorders, One Common Therapeutic Target
Maurizio Pacifici, PhD Children’s Hospital of Philadelphia