Title: Evaluating gene therapies for Pompe disease - clinical trial goals and assessments

Date: Monday, February 26, 2024

Speakers:
Jordi Díaz-Manera, MD, PhD
Christine S. Brown
Mark Walzer, PhD

Webinar Overview:
This Astellas Gene Therapies presentation provides an overview of the different gene therapy approaches for late-onset Pompe disease (LOPD) currently under evaluation in clinical trials, including liver-directed, central nervous system (CNS)-directed, and muscle-directed. The speakers will explain how clinical trials help determine whether a potential gene therapy is a viable treatment option for people living with LOPD. They will also help the audience understand the goals of clinical trials and the assessments that measure those goals.

Speaker Bios:

Jordi Díaz-Manera, MD, PhD - Professor of Neuromuscular Diseases at Newcastle University
Dr. Jordi Díaz-Manera is a Professor of Neuromuscular Disorders, Translational Medicine and Genetics at Newcastle University. His research has focused on understanding the process of muscle degeneration and regeneration in people living with neuromuscular diseases, including Pompe disease, and developing new diagnostic tools and therapies for muscle diseases.

Pompe Disease is one of Professor Díaz-Manera's main interests. His findings have helped improve its diagnosis, he has contributed to the development of treatment guidelines for the disease and is a member of the Steering Committee of the European Pompe Consortium (EPOC).

Professor Díaz-Manera has received a fee from Astellas for his time spent preparing for and participating in this webinar.

Christin S. Brown - Associate Director, Patient Partnerships, Patient Centricity at Astellas
Christine S. Brown's role at Astellas is focused on building trusted relationships with patient communities to support improved patient outcomes. Christine has worked on all sides of medicines development, including the Global Genes RARE-X research program, the Pfizer Rare Disease Research Unit, and as the Executive Director of the National PKU Alliance. Christine is passionate about bringing patient experiences to clinical trial design.

Christine was a member of the FDA Patient Engagement Collaborative and a subcommittee member the Secretary's Advisory Council on Heritable Disorders in Newborns and Children. She also received the 2021 Rare Voice Award for federal patient advocacy from the Everylife Foundation.

Mark Walzer, PhD - Director, Clinical Development at Astellas Gene Therapies
Dr. Mark Walzer has worked in pharmaceutical clinical research and development for nearly 20 years and currently lads clinical development activities for Astellas Gene Therapies programs. Mark joined Astellas in 2013, focusing on biomarker and early-stage clinical development for small molecules and biologics in the Neuroscience, Pain and Infectious Diseases areas. More recently, he has been directing clinical strategy to support multiple early-stage AAV-based gene and cell therapy programs in Ophthalmology and metabolic muscle disorders, including Pompe disease. Prior to his time at Astellas, Mark lead various nonclinical and clinical research activities at Ovation Pharmaceuticals and Lundbeck focusing on rare diseases in the Neuroscience space.

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