CRANBURY, N.J., May 06, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (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, 4:45-5:00 p.m. ET
- 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.
About Batten Disease
Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these diseases, a defect in a specific gene triggers a cascade of problems that interferes with a cell’s ability to recycle certain molecules. Each gene is called CLN (ceroid lipofuscinosis, neuronal) and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.
Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding, and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment, and seizures. Patients typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden, and die prematurely.
About Amicus Therapeutics
Amicus Therapeutics (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 www.amicusrx.com, and follow us on Twitter and LinkedIn.
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans or projections will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical products; and the potential that we will need additional funding to complete all of our studies and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2019. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
Director, Investor Relations