UPDATE – Amicus Therapeutics Announces Upcoming Presentations at the American Society of Gene & Cell Therapy 23rd Annual Me...
May 06 2020 - 7:00AM
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, 4:45-5:00 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 DiseasePompe
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 DiseaseBatten
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 (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 www.amicusrx.com, and follow us on Twitter and
LinkedIn.
Forward-Looking StatementsThis
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.
CONTACTS:
Investors/Media:Amicus
TherapeuticsAndrew FaughnanDirector, Investor
Relationsafaughnan@amicusrx.com(609) 662-3809
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