The Alliance for Regenerative Medicine Announces Inherited Blood Disorders Clinical and Patient Education Roundtable

– November 17, 2016

WASHINGTON, D.C., November 17, 2016 – The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the gene and cellular therapies and broader regenerative medicine sector, announced it will hold its Inherited Blood Disorders Clinical and Patient Education Roundtable December 13 in Washington, D.C., the third event in its patient roundtable series.

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.

Held in partnership with the Hemophilia Federation of America and the National Hemophilia Foundation, and sponsored by Shire, Sangamo BioSciences and uniQure, this event will focus on educating multi-sector stakeholders about the latest scientific progress and delivery challenges associated with hemophilia and other inherited blood disorders.

“Many gene and cell therapies developers are advancing transformative approaches to treating and potentially curing inherited blood disorders, including hemophilia, beta-thalassemia, sickle cell disease and more,” said Morrie Ruffin, ARM managing director. “We are pleased to serve an educational role to provide patients with information on the latest advances and potential impact of this technology, especially as several therapies are moving through late-stage clinical evaluation.”

This roundtable discussion is expected to attract more than 100 individuals, including patient advocates, industry leaders and clinicians.

Preliminary Roundtable Agenda:

1:30 – 1:40pm       Welcome & Introductory Remarks
Kimberly Haugstad
President & CEO
Hemophilia Federation of America

1:40 – 2:10pm       Featured Talk: Gene Therapy 101 and Clinical Outlook
Steven Pipe, M.D.
Director, Division of Pediatric Hematology & Oncology
Pediatric Medical Director, Hemophilia & Coagulation Disorders Program
University of Michigan

2:10 – 3:05pm       Roundtable Discussion: State of the State of Gene Therapy for Inherited Blood Disorders
Daniel Leonard, Director of Global Patient Advocacy, uniQure
Didier Rouy, M.D., Medical Director, Hemoglobinopathies, Sangamo BioSciences

3:05 – 3:20pm       Break

3:20 – 4:15pm       Roundtable Discussion: Clinical Trial Recruitment and Interaction with Patient Community
John Chapin, M.D.
Medical Director, Clinical Research and Development – Hematology

Sudha Parasuraman, M.D.
Vice President of Global Medical Affairs

4:15 – 4:45pm       Featured Talk: The Patient’s Perspective
John Vieke
Patient Advocate

4:45 – 5:00pm       Closing Remarks: Next Steps
Val Bias
National Hemophilia Foundation

5:00 – 6:00pm       Networking & Cocktail Reception

This preliminary agenda is available online, with additional details to be added over the coming weeks.

This event will take place at the Carnegie Library at Mt. Vernon Square, 801 Mt. Vernon Square NW, Washington, D.C. 20001.

Registration is now open. The cost of attendance is $150 for industry attendees; $75 for non-profit and government attendees. The event is complimentary for patients, patient advocates, clinician attendees and members of the media. For more information and to register, please visit the event’s webpage.

For questions regarding this event, please contact ARM’s Patient Advocacy & Events Manager Chelsey Hathaway, For media interested in attending this event, please contact ARM’s Senior Director of Communications Lyndsey Scull,

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 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit