Gene Therapy for Rare Diseases: Strategies to Drive Operations

Life Sciences, Clinical Trials, Drug Discovery & Development, Cell and Gene Therapy,
  • Wednesday, February 10, 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.

At the highest level, clinical development in a timely manner relies upon:

  • Partnering with the patient community at the earliest stages
  • Partnering with regulators and clinical research sites
  • Appropriately partnering with study participants and their families, especially in consideration of their relative distance from the treatment centers and the duration of long-term follow-up

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.


Amy Raymond, PRA Health Sciences

Amy Raymond, PhD, PMP, Director of Therapeutic Expertise, Center for Rare Diseases, PRA Health Sciences

Amy has a comprehensive understanding of drug discovery and drug development from the research laboratory to patient bedsides from 20+ years of experience in academic institutions, pharmaceutical companies, and CROs. She earned her doctorate in molecular and cellular biology and received postdoctoral training at Memorial Sloan-Kettering Cancer Center. She has contributed to the discovery and development of new treatments in a wide array of indications, from rare cancers to development of gene therapy for rare neurodegenerative disorders. Amy leads PRA’s Rare Disease Gene Therapy Working Group and serves as a subject matter expert within the Center for Immuno-oncology, Cellular and Gene Therapy. In her role at PRA Health Sciences, Amy collaborates with drug development partners to optimize development programs and remains actively involved throughout execution to support program success.

Message Presenter
Chris Jenkins, Sabai Global

Chris Jenkins, PhD, MPH, RBP, CHMM, Chairman of Sabai Global

Chris has advanced degrees in biology, public health, and biosecurity from the University of Missouri and Saint Louis University. He has served as a biosafety officer at multiple Tier 1 Institutions, served as an SVP with a leading clinical services organization, WIRB-Copernicus Group (WCG), as well as run Research Compliance at the University of Texas at Austin. He serves on multiple advisory boards to gene and cell therapy organizations, and Sabai Global supports over 200+ sponsors on over 200+ unique gene and cell therapy protocols in the US and ex-US.

Message Presenter
Christine Waggoner, Cure GM1 Foundation

Christine Waggoner, President and Co-Founder, Cure GM1 Foundation

For Christine Waggoner, founding and running Cure GM1 is a true labor of love to help bring a treatment to all those affected by GM1 gangliosidosis. Rare diseases affect 1 in 10 people and there is no other 501(c)(3) entirely dedicated to GM1. Cure GM1 funds medical research for GM1 gangliosidosis, such as AAV gene therapy, enzyme replacement therapy, chaperones, inhibitors and substrate reduction therapies. Cure GM1 has raised ~$3.3M+ to date through social media and grassroots fundraising.

Message Presenter

Who Should Attend?

This webinar will appeal to VPs, Directors, Managers, Department Heads, Scientists and Researchers working within:

  • Clinical Affairs
  • Clinical R&D
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Operations
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

What You Will Learn

In this webinar, participants will learn about:

  • The scientific, clinical, regulatory and operational complexities specific to rare disease clinical development
  • How to understand and operationalize the stakeholder dynamics unique to each patient population and treatment type
  • Strategies for the successful development of gene therapies

Xtalks Partner

PRA Health Sciences

PRA Health Sciences delivers innovative drug development solutions that improve patients’ lives , including our proven commitment to developing the gene therapies for rare disease patient communities. Our people are passionate about clinical research, working tirelessly to provide quality results and share our partners’ mission to develop advanced therapeutics to address unmet needs. We offer exceptional experience across all phases and therapeutic areas and a broad spectrum of solutions, ranging from full-service clinical development to a pioneering embedded model.

With 16,000+ employees covering 90+ countries, we bolster an impressive global presence with keen local insights. Our project teams harness their understanding of local regulations, standards of care and cultural customs to effectively align our approaches with each study’s unique goals.

At PRA, we love what do because we are making a difference in the lives of patients and their family members worldwide by driving development of meaningful treatments. Over the years, we have contributed to the development of numerous drugs now available to countless patients. From our scientific and medical experts to therapeutically aligned project managers and monitors, we provide the commitment and expertise needed for today’s complex studies.

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