2023

Philip J. (PJ) Brooks is the acting director of NCATS’ Division of Rare Diseases Research Innovation. 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.

Michele Herndon is the co-founder and vice-president of the Mitchell and Friends Foundation.  The foundation raises awareness of Mitchell Syndrome, promotes research into possible treatments, and offers support to patients and families affected by Mitchell Syndrome.  This newly discovered disease was discovered in part through information learned from Michele’s oldest son, Mitchell, who was the first patient identified.  Professionally, Michele has been a pediatric nurse since 2005 and is currently a nursing manager for St. Louis Children’s Hospital in pediatric trauma and injury prevention.

Dr. Edward Neilan is the Chief Medical and Scientific Officer at the National Organization for Rare Disorders (NORD®). Ed is a molecular biologist, pediatrician, and medical and biochemical geneticist. He has also worked on many rare disease clinical trials and has contributed to the clinical development and FDA-approval of several new rare disease therapies. Immediately prior to joining NORD, Ed worked at Sanofi Genzyme, where he led global medical affairs for rare neurogenetic diseases and also contributed to clinical development efforts across multiple programs. Prior to that, Dr. Neilan served a term as President of the Medical Staff at Boston Children’s Hospital. In addition to his full-time role at NORD, Dr. Neilan also still maintains part-time faculty appointments at Boston Children’s Hospital and Harvard Medical School.

Tracy is involved with advocating for all rare diseases within various agencies and with members of Congress. Tracy has spoken before the FDA on multiple occasions, been invited as a panelist for cell based therapies at the NHLBI within the NIH, and has been a featured panelist at a Rare Disease Congressional Caucus hearing on Capitol Hill.  Over the years Tracy & Jennifer have worked with many other patient advocates to advance legislation including the 2012 FDASIA bill and the 2016 21^st Century Cures Act.