Treatment News

Amicus Therapeutics to Present Preclinical Pompe Disease Gene Therapy Data at the American Society of Gene & Cell Therapy ASGCT 22nd Annual Meetin

3.0 from 2 votes
Monday, April 15, 2019

Learn more about:

Access Programs

CRANBURY, N.J., - Amicus Therapeutics (Nasdaq: FOLD) today announced the company will present initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Pompe disease in a poster at the American Society of Gene & Cell Therapy (ASGCT) 22nd Annual Meeting on April 30, 2019 in Washington D.C.

Poster Presentation:

  • Title: “Development of a Novel Gene Therapy for Pompe Disease: Engineered Acid Alpha-Glucosidase Transgene for Improved Expression and Muscle Targeting”
  • Session Date and Time: Tuesday, Apr 30, 2019, 5:00 PM - 6:00 PM ET
  • Session title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases
  • Room: Columbia Hall
  • Abstract number: 518

These represent the first set of preclinical results for the Pompe gene therapy which Amicus is currently developing in collaboration with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania (Penn).  This relationship combines Amicus’ protein engineering and glycobiology expertise with Penn’s adeno associated virus gene transfer technologies to develop AAV gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability.
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA leads to accumulation of glycogen in cells, which results in the clinical manifestations of Pompe disease. The disease can be debilitating, and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.

About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information, please visit the company’s website at, and follow us on Twitter and LinkedIn.


Amicus Therapeutics
Sara Pellegrino, IRC
Vice President, Investor Relations & Corporate Communications
(609) 662-5044

Amicus Therapeutics
Marco Winkler
Director, Corporate Communications
(609) 662-2798

Source: Amicus Therapeutics
3.0 from 2 votes
Free Newsletter
Related Videos
by Scott Harwood
by Scott Harwood