Check Back Soon for Upcoming Webinars

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.

Title: Spark Therapeutics Webinar: Trail Expectations – What to Expect
Date: Wednesday, September 27, 2023
Time: 1 PM CT / 2 PM ET
Speaker:
Jacose Bell
Webinar Overview:
This presentation, developed by Spark Therapeutics, takes audience members through the introductory science of AAV gene therapy. We will examine how AAV theoretically works in a liver-directed approach, how that is different from other investigational forms of cell and gene therapy, and what the challenges and potential risks are with AAV gene therapy. The presentation will then focus the remainder of its time on the role of clinical research and things people should consider if they are interested in participating in clinical research for investigational gene and cell therapies.

Title: New insights into Pompe disease since the advent of NBS
Date: Thursday, August 10, 2023
Time: 1 PM CT / 2 PM ET
Speakers:
Dr. Priya Kishnani, MD
Erin Huggins, MS, CGC
Webinar Overview:
An overview of the lessons learned from newborn screening for Pompe disease and its impact on disease management.