Webinar Details
Title: Lentiviral gene therapy for Pompe disease
Date: Tuesday, October 10, 2023
Time: 10 AM CT, 11 AM ET
Speaker:
Dr. Pim Pijnappel, Associate Professor in Cell and Gene Therapy at the Erasmus MC University Medical Center, Rotterdam, the Netherlands
Webinar Overview:
The webinar will start with an introduction on Pompe disease with the latest information on what we know of the disease and what the current challenges are for treatments. Then, the concept of gene therapy will be explained as a possible new treatment option. The focus will be on lentiviral gene therapy, which is 1 of many possible forms of gene therapy. Recent results from the laboratory will be presented, followed by an update on next steps toward clinical development. The current landscape of new therapies for rare diseases will be discussed, including the need to develop novel therapies for reasonable and transparent prices.
Speaker Biography
Dr. Pim Pijnappel
Associate Professor in Cell and Gene Therapy, Erasmus MC University Medical Center, Rotterdam, The Netherlands
Dr. W.W.M. Pim Pijnappel is associate professor at the Erasmus MC and has a broad expertise ranging from basic to clinical science. He performed his PhD research at the Hubrecht Laboratory (Utrecht, the Netherlands), and post-doctoral research at the EMBL Heidelberg and TU Dresden (Germany). His research group at the Center for Lysosomal and Metabolic Diseases at Erasmus MC in Rotterdam, the Netherlands, focuses on basic, translational, and clinical aspects of lysosomal storage disorders including Pompe disease and Hunter syndrome. The aim of his research is to obtain a better understanding of disease mechanisms, to develop novel treatment options, and to understand genotype/phenotype relationships. He has published >80 peer reviewed scientific articles and holds 11 patents on therapies and biomedical technology.