Title: Lentiviral gene therapy for Pompe Disease

Date: Tuesday, October 10, 2023

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 Bio:
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.
Website:
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