Amicus Therapeutics Enters Research and Development Collaboration with University of Pennsylvania to Develop AAV Gene Therapi...
08 October 2018 - 10:00PM
Collaboration Focuses on Four Genetic Disorders:
Pompe, Fabry, CDKL5 and One Additional Undisclosed Rare Metabolic
Disorder
Amicus Therapeutics (Nasdaq: FOLD) today announced a major
collaboration with the Gene Therapy Program in the Perelman School
of Medicine at the University of Pennsylvania (Penn) to pursue
research and development of novel gene therapies for Pompe disease,
Fabry disease, CDKL5 deficiency and one additional undisclosed rare
metabolic disorder. This relationship will combine Amicus’ protein
engineering and glycobiology expertise with Penn’s adeno associated
virus (AAV) gene transfer technologies to develop AAV gene
therapies designed for optimal cellular uptake, targeting, dosing,
safety and manufacturability.
“This groundbreaking collaboration with Penn offers a new
opportunity to potentially transform the lives of people living
with these severe genetic disorders,” stated John F. Crowley,
Chairman and Chief Executive Officer of Amicus. “For people living
with Fabry, it is a fulfillment of our pledge to advance science
toward a cure for Fabry disease. We are partners now with the Fabry
community for life. For Pompe, this is another important step on a
lifelong journey toward the ultimate answer to finally cure Pompe.
If we are to do so, we must have a gene therapy that addresses the
many technical challenges in Pompe disease and that employs state
of the art science in both protein and gene therapy engineering.
For CDKL5 and other rare metabolic disorders, this collaboration is
the foundation for advancing new therapies for these devastating
genetic disorders. Dr. Wilson’s laboratory and The Gene
Therapy Program at Penn has strong capabilities in its AAV vector
technology, as well as manufacturing and immunology strategies to
develop novel gene therapy candidates, and to successfully advance
them through preclinical development. This is another important
step in Amicus becoming the leader in gene therapy for rare
metabolic disorders.”
Penn’s AAV vector technology is designed to improve targeting,
tropism, safety, immunogenicity, and gene delivery, while Amicus’
protein engineering capabilities may optimize protein expression,
secretion, targeting and uptake of the target protein. The
agreement between Amicus and Penn is a Research, Collaboration and
License Agreement, with funding provided to Penn to advance the
preclinical research programs in the Wilson Lab and to license
certain technologies invented under the funded Research
Collaboration. The collaboration program will focus on developing
innovative new AAV gene therapies for Pompe disease, Fabry disease,
CDKL5 deficiency and one additional undisclosed rare metabolic
disorder.
“Amicus has developed unique abilities in drug development in the
lysosomal storage disorders, particularly in Pompe and Fabry
diseases,” said James M. Wilson, MD PhD, Professor of Medicine and
Pediatrics at the Perelman School of Medicine. “There are multiple
and unique challenges in developing optimal gene therapy products
for patients living with Pompe and Fabry diseases. I believe that
we can combine the technologies and capabilities from my research
laboratory at Penn with the Amicus expertise in protein
engineering, glycobiology and disease biology understanding to
rapidly advance novel gene therapies to the clinic. Because of
their unique capabilities and their commitment to great science and
to patients, we are excited about our new partnership with Amicus
to develop AAV gene therapies for patients with urgent unmet
needs.”
Additional details and an overview of the collaboration will be
provided during the Amicus Analyst Day on October 11, 2018.
About Amicus TherapeuticsAmicus Therapeutics
(Nasdaq: FOLD) is a global, patient-centric 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.
Forward Looking StatementThis press release
contains "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995 relating to the
collaboration with the University of Pennsylvania, and the
development of potential gene therapy product candidates. The
inclusion of forward-looking statements should not be regarded as a
representation by us that any of our plans 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, the benefits of this collaboration may never be realized,
the potential that results of clinical or preclinical studies
indicate that the product candidates are unsafe or ineffective; the
potential that it may be difficult to enroll patients in our
clinical trials; the potential that regulatory authorities,
including the FDA, EMA, and PMDA, may not grant or may delay
approval for our product candidates; 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 or commercial
products; the potential that we will need additional funding to
complete all of our studies and manufacturing and the potential
that certain individuals may not continue to support the
development of product candidates. 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, 2017 as well as our
Quarterly Report on Form 10-Q for the quarter ended June 30, 2018
filed August 7, 2018 with the Securities and Exchange Commission.
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 presentation to reflect events or circumstances
after the date hereof.
CONTACTS:
Investors/Media:Amicus TherapeuticsSara
Pellegrino, IRCVice President, Investor Relations & Corporate
Communicationsspellegrino@amicusrx.com (609) 662-5044
Media / Multimedia Assets:Pure
CommunicationsJennifer
Paganellijpaganelli@purecommunications.com (347) 658-8290
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