Amicus Therapeutics Announces Upcoming Presentations at the American Society of Gene & Cell Therapy 23rd Annual Meeting

CRANBURY, N.J., May 05, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases today announced the acceptance of several abstracts for presentation at the American Society of Gene & Cell Therapy 23rd Annual Meeting being held virtually on May 12 – 15. Preclinical data from its Pompe gene therapy program, which Amicus is developing with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania, will be presented as an oral presentation. Preclinical data related to the CLN6 and CLN8 Batten disease programs, with our partners at Sanford Research and Nationwide Children’s Hospital, will be presented in respective posters.

Oral Platform Presentation: Thursday, May 14, 2020, 5:15-5:30 p.m. ET

Pompe Disease:

• Title: “Combined CNS and Systemic Directed Gene Therapy in a Mouse Model of Pompe Disease with Advanced Disease at Treatment”
• Session title: Musculoskeletal Diseases II
• Abstract number: 929
• Presenter: Juliette Hordeaux, DVM, PhD, Senior Research Director, Translational Research, Gene Therapy Program, University of Pennsylvania

Poster Session: Tuesday, May 12, 2020, 5:30-6:30 p.m. ET

CLN6 Batten Disease:

• Title: “Intracerebroventricular Delivery of AAV9 Gene Therapy Prevents Central and Retinal Neuron Loss, Preserving Vision in CLN6-Batten Disease Mice”
• Session title: Neurologic Diseases
• Abstract number: 303

Poster Session: Wednesday, May 13, 2020, 5:30-6:30 p.m. ET

CLN8 Batten Disease:

• Title: “Neonatal Administration of scAAV9. CLN8 Gene Therapy Prevents Disease Pathology and Behavioral Deficits in a Mouse Model of CLN8-Batten Disease”
• Session title: Pharmacology/Toxicology Studies or Assay Development
• Abstract number: 896

All abstracts for the American Society of Gene & Cell Therapy 23rd Annual Meeting are now available online.

About Pompe Disease
Pompe disease is an inherited lysosomal 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.