Dr. Crooke is the founder, chief executive officer and chairman of n-Lorem Foundation. He’s also the founder, former chairman and chief executive officer of Ionis Pharmaceuticals, the leader in RNA-targeted therapeutics. Under Dr. Cooke’s leadership, Ionis pioneered development of the revolutionary antisense technology platform and created one of the largest, most advanced therapeutic pipelines in the biotechnology industry.

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.

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.

After being diagnosed with a rare brain tumor, NF2, Onno Faber founded Rarebase, a public benefit precision medicine company that leverages cutting-edge technology and biology to discover and develop treatments for millions of people worldwide living with a rare disease.  He is dedicating his life to create new avenues for rare disease R&D ultimately giving everyone with a rare disease the opportunity to leverage the technology for a healthy life.

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.