WASHINGTON, D.C., January 20, 2016 – The Alliance for Regenerative Medicine (ARM), the international organization representing the gene therapy, cell therapy and tissue engineering sectors, announced the first event in its patient advocacy roundtable series, the Rare Disease Clinical and Patient Education Roundtable, to be held January 27, 2016 in Washington, D.C.
ARM’s patient roundtable series is intended to serve as an educational resource for the broader patient community and to help bridge the gap between industry and patient groups who are working towards the common goal of durable, and potentially curative, therapies.
This first event will focus on educating multi-sector stakeholders about the latest scientific progress and delivery challenges associated with rare diseases, specifically as they relate to regenerative medicine. This roundtable discussion is expected to attract nearly 100 individuals, including patient advocates, industry leaders and clinicians.
Program Agenda:
2:00 – 2:15pm Welcome & Introductory Remarks
Ron Bartek, President & Co-Founder, Friedreich’s Ataxia Research Alliance
Morrie Ruffin, Managing Director, Alliance for Regenerative Medicine
2:15 – 2:45pm Featured Talk
Barry Byrne, M.D., Ph.D., Director, Powell Center for Rare Disease Research and Therapy, University of Florida
2:45 – 3:45pm Research for Rare Disease: Unique Opportunities and Challenges
Julie Venners Christensen, Head of Global Patient Advocacy – Gene Therapy R&D, Rare Disease Unit, GlaxoSmithKline
Sharon Hesterlee, Ph.D., Chief Science Officer, Myotonic Dystrophy Foundation
Karen Kozarsky, Ph.D., CEO, Vector Partners
3:45 – 4:00pm Afternoon Break
4:00 – 5:00pm Collaborating on Cures for Rare Disease
Ron Bartek, President & Co-Founder, Friedreich’s Ataxia Research Alliance
Jennifer Cutie, M.D., Associate Director, Patient Advocacy, Voyager Therapeutics
Tim Miller, Ph.D., President & CEO, Abeona Therapeutics
Barbara Wuebbels, Vice President of Patient Advocacy, Audentes Therapeutics
5:00 – 5:30pm Featured Talk: The Patient Perspective
Michele Rhee, Head of Global Patient Affairs, bluebird bio
Jean Walsh, Patient Advocate; Ambassador, Friedeich’s Ataxia Research Alliance
5:30 – 5:45pm Closing Remarks: Next Steps
Michael Werner, Executive Director, Alliance for Regenerative Medicine
5:45 – 7:00pm Networking & Cocktail Reception
“Regenerative medicine and other advanced therapies hold immense promise for rare disease patients,” said Michael Werner, executive director of the Alliance for Regenerative Medicine. “Numerous companies active in this sector are developing transformative treatments and potential cures, focusing on the root cause of disease and disorders, not solely the symptoms.”
The event will take place at the Carnegie Library – 801 K Street NW, Washington, D.C. 20001.
Cost of attendance is $125 for industry attendees; $75 for non-profit and government attendees and complimentary for patients and clinician attendees. For more information and to register, please visit http://alliancerm.org/event/arms-rare-disease-roundtable.
About The Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 230 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.
