July 18 webinar delves into gene therapy trials for rare diseases
Challenges in running studies, need for patient voice in design, among topics
A free webinar on July 18 will offer insights into the patient journey in gene therapy trials, with a particular focus on rare diseases such as AADC deficiency.
The webinar, “Rare Disease Trials and Gene Therapy: Understanding the Patient Journey,” will broadcast live beginning at 10 a.m. EDT, and explore various aspects of gene therapy trials and research, according to a press release announcing the event.
Offered by Thermo Fisher Scientific’s PPD clinical research business and hosted by Xtalks, it largely is directed at patient advocacy and study groups bringing clinical research closer to communities, companies involved in trial sites and patient recruitment, and health professionals involved in clinical research, especially for rare diseases. Those interested in attending are required to register.
Several gene therapies, like Upstaza, approved in recent years for rare diseases
Gene therapy, which generally works by providing cells with a working copy of a gene whose mutations cause disease, has gained increasing interest in recent years. Several gene therapies have been approved for certain rare genetic diseases.
Recently, Upstaza (eladocagene exuparvovec) became the first gene therapy approved in Europe, including the U.K, for AADC deficiency in patients starting at 18 months of age, and the first to target the disease’s underlying mechanism.
PTC Therapeutics, which is developing the therapy, plans to apply to the U.S. Food and Drug Administration for its approval in the coming months.
Webinar attendees will learn more about gene therapy and its importance to rare disease patients, as well as challenges that scientists face in running gene therapy trials, including therapy manufacturing, patient recruitment, site selection and activation, and regulatory considerations.
The webinar “also will shed light on the importance of incorporating patient feedback into [trial] protocol design, recognizing it as a critical step in advancing these therapies,” the release stated.
The webinar will feature speakers with PPD, a part of Thermo Fisher Scientific, including:
- Meagan Spychala, the executive director of PPD’s project management, with experience overseeing the operations of rare disease clinical trials and trials testing gene and cellular therapies;
- Kim Watanabe, PhD, the executive director of cell and gene therapy at PPD, whose focus is on research, development, and the operational strategy of cell and gene therapies;
- Panteli Theocharous, PhD, PPD’s global vice president and cell and gene therapy strategy lead, with expertise in cancer immunotherapy.
In addition to previously mentioned topics, the speakers will address the importance of long-term follow-up in gene therapy studies for rare diseases, as well as the need to use optimized digital technologies.
Xtalks, powered by Honeycomb Worldwide, provides educational webinars to the global life science, food, and medical devices communities. Thermo Fisher Scientific offers products and services to support companies working in treatment development, including for cell and gene therapies.
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