Just as the upstream research and development of these new treatment modalities required a new kind of toolkit, successfully developing these gene therapies benefits from understanding and operationalizing the stakeholder dynamics unique to the patient population and treatment type.
TORONTO (PRWEB) January 18, 2021
There are approximately 7,000 distinct rare diseases affecting 350 million people worldwide, but only 5 to 10 percent of rare diseases have an FDA-approved treatment. Because approximately 80 percent of rare diseases have a known monogenic cause, continued advancements in gene replacement and genome editing therapies offer remarkable hope for durable disease-modifying treatments.
There are scientific, clinical, regulatory and operational complexities specific to the interface of rare disease clinical development and development of gene therapies. Just as the upstream research and development of these new treatment modalities required a new kind of toolkit, successfully developing these gene therapies benefits from understanding and operationalizing the stakeholder dynamics unique to the patient population and treatment type.
These challenges include a wide geographic distribution of a relatively small patient population in each rare disease, with potential heterogeneity amongst patients and attendant challenges of defining natural history; balancing the volume of data to be collected against making the study achievable for sites and participants; and longer start-up timelines to satisfy regional and local regulatory bodies.
Register for this webinar to join a cross-enterprise conversation on strategies to drive operations for the successful development of gene therapy for rare diseases.
Join Amy Raymond, PhD, PMP, Director of Therapeutic Expertise, Center for Rare Diseases, PRA Health Sciences; Chris Jenkins, PhD, MPH, RBP, CHMM, Chairman of Sabai Global; and Christine Waggoner, President and Co-Founder, Cure GM1 Foundation, in a live webinar on Wednesday, February 10, 2021 at 11am EST (4pm GMT/UK).
For more information, or to register for this event, visit Gene Therapy for Rare Diseases: Strategies to Drive Operations.
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