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

WASHINGTON, DC--(Marketwired - Nov 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 these technologies, 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