Dr Lutz, Ph.D., M.B.A. is the Vice President of the Rare Disease Translational Center at The Jackson Laboratory (JAX). With 25 years of experience in mouse genetics, Dr. Lutz has focused her research efforts on patient organizations and families faced with a rare disease diagnosis. The JAX Rare Disease Translational Center incorporates precision mouse models and broad-based drug efficacy testing to support Investigational New Drug (IND) enabling studies.

David Fajgenbaum, MD, MBA, MSc, is an Associate Professor at the University of Pennsylvania and Founding Director of the Center for Cytokine Storm Treatment & Laboratory. He also serves as Associate Director of Patient Impact at Penn Orphan Disease Center and is the Co-Founder and President of the Castleman Disease Collaborative Network. A patient himself battling idiopathic multicentric Castleman disease (iMCD), Dr. Fajgenbaum is the author of the bestselling book Chasing My Cure and has identified numerous treatment approaches for iMCD and cancer. He recently co-founded Every Cure, a nonprofit dedicated to unlocking the potential of FDA-approved drugs to treat a range of diseases.

Pat Furlong is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States focused solely on Duchenne muscular dystrophy (Duchenne). Its mission is to improve the treatment, quality of life, and long-term outlook for individuals affected by Duchenne through research, advocacy, education, and compassion. Duchenne is the most common fatal, genetic childhood disorder, which affects approximately 1 out of every 5,000 boys each year worldwide. It currently has no cure.

Dr. Kakkis is Chief Executive Officer, President and a Director of Ultragenyx. He founded Ultragenyx in 2010 to create a company that is uniquely built around a deep and meaningful engagement with rare disease patients and their caregivers to fully understand their needs.  The Ultragenyx approach includes designing a novel development model that fundamentally changes the established paradigms, as well as challenges traditional beliefs around access to therapies for patients with rare and ultra-rare genetic diseases.

Jim Geraghty is an industry leader with over 35 years of strategic leadership experience, including more than 25 years as a senior member of executive teams at biotechnology companies developing and commercializing innovative therapies. Mr. Geraghty was previously  an Entrepreneur in Residence at Third Rock Ventures, a leading biotech venture fund, Sanofi S.A., and Genzyme Corporation.  His book, Inside The Orphan Drug Revolution tells the difficult history of drug treatment for rare diseases.

Philip J. (P.J.) Brooks is the acting director of NCATS’ Division of Rare Diseases Research Innovation. Mr. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project and the International Rare Diseases Research Consortium (IRDiRC). He also is the working group co-coordinator for the NIH Common Fund program on Somatic Cell Genome Editing, one of the leaders of the Platform Vector Gene Therapy (PaVe-GT) pilot project and the co-chair of the Bespoke Gene Therapy Consortium.

Dr. Gray’s core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system.  His major focus is in AAV vector development to create vectors tailored to serve specific clinical and research applications involving the nervous system.  These include the development of novel AAV capsids amenable to widespread Central Nervous System (CNS) gene transfer.  As AAV-based platform gene transfer technologies have been developed to achieve global, efficient, and in some cases cell-type specific CNS gene delivery, his research focus has also included preclinical studies to apply these reagents toward the development of treatments for neurological diseases.

Wendy Chung, MD, PhD is a clinical and molecular geneticist and the Chief of the Department of Pediatrics at Boston Children’s Hospital and Harvard Medical School. Dr. Chung directs NIH funded research programs in human genetics of pulmonary hypertension, breast cancer, obesity, diabetes, autism, birth defects including congenital diaphragmatic hernia and congenital heart disease.

Dr. Chung is a national leader in the ethical, legal, and social implications of genomics. She was the recipient of the Rare Impact Award from the National Organization of Rare Disorders, and is a member of the National Academy of Medicine and the American Academy of Physicians. Dr. Chung received her B.A. in biochemistry from Cornell University, her M.D. from Cornell University Medical College, and her Ph.D. from The Rockefeller University in genetics.

Peter Marks, M.D., Ph.D. is the director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration. The center is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies.

Dr. Marks and center staff are committed to facilitating the development of biological products and providing oversight throughout the product life cycle.