Laboratory news & BioScience March 2017

NEW ZEALAND LABORATORY NEWS | NEW ZEALAND BIOSCIENCE
VOL. 26 NO. 1 | MARCH 2017
Orchard Therapeutics and PharmaCell
B.V. announce manufacturing alliance
London, UK & Maastricht, the
Netherlands -- Orchard Therapeutics
(“Orchard”), a clinical-stage
biotechnology company dedicated to
bringing transformative ex-vivo gene
therapies to patients with serious
and life-threatening orphan diseases
recently announced an alliance with
PharmaCell B.V. (“PharmaCell”), a
leading Contract Manufacturing
Organisation (CMO) for Cell and Gene
Therapies and Regenerative Medicine.
Under the terms of the alliance, Pharma-
Cell will provide GMP-compliant manufacturing
services to support clinical trials
and commercialization of Orchard’s ex-vivo
autologous gene therapy products.
This agreement represents another important
milestone for Orchard’s strategy to establish
a global supply chain to deliver ex-vivo autologous
gene therapy medicines to patients
with devastating genetic diseases.
Stewart Craig, Ph.D. Orchard’s chief manufacturing
officer commented: “We are delighted
to partner with PharmaCell, a world-leading
CMO with a proven track record in the manufacture
and supply of cell-based products for
both clinical trials and commercial markets. We
are excited about the prospect for this partnership
to accelerate our plans to make medicines
available on a global basis.”
Orchard’s clinical development pipeline includes
novel treatments for primary immune
deficiency disorders and inherited metabolic
disorders, including ADA-SCID (adenosine deaminase
deficiency severe combined immunodeficiency)
and MPS-IIIA (Mucopolysaccharidosis
IIIA or Sanfilippo syndrome type A) as well as
other indications.
Alexander Vos, chief executive officer of
PharmaCell, commented: “We are very excited
to have the opportunity to collaborate with Orchard
Therapeutics on their ground-breaking
development pipeline for severely debilitating
genetic disorders. PharmaCell will bring its
extensive resources and experience to bear to
support the global commercialization of these
innovative therapies like we have been and are
doing for an increasing portfolio of late-stage
clinical programs for leading industry players."
About adenosine deaminase deficiency
severe combined immunodeficiency
(“ADA-SCID”)
ADA-SCID is a rare inherited disorder of
the immune system. The incidence of ADA-
SCID is estimated between 1 in every 375,000
to 660,000 live births, according to literature
sources. ADA-SCID is caused by mutations in
the gene encoding for the enzyme adenosine
deaminase, which result in a severe deficiency
PRODUCT NEWS
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in white blood cells and life-threatening infections.
In the absence of treatment, ADA-SCID
is fatal within the first months of life. Despite
currently available treatment options, there
remains significant need for therapies that reduce
the mortality, morbidity and burden of
disease on patients and families.
About mucopolysaccharidosis type IIIA
(MPS IIIA, Sanfilippo syndrome type A)
MPS IIIA is a rare neurodegenerative lysosomal
storage disease caused by mutations in
the sulfoglycosamine sulfohydrolase (SGSH)
gene. There are no effective treatments for MPS
IIIA to date. The disease affects children in early
life, with a progressive decline in cognitive and
behavioural function and a subsequent decline
in motor function and results in severe dementia
and early death, usually in the teens or early
twenties.
About PharmaCell B.V.
PharmaCell is a leading European-based
CMO exclusively focused in the area of cell
and gene therapy, and regenerative medicine.
PharmaCell has experience in supporting
Phase I through Phase III clinical trials
and early commercial manufacturing in cell
and gene therapy in terms of manufacturing,
Quality Control, storage, in-outgoing logistics
and product release through its in-house
QPs. Its services also include process and
assay-development to ensure GMP compliance,
robustness and scalability of cell therapy
manufacturing processes. PharmaCell offers a
unique manufacturing platform in Europe to
support the growth of the cell therapy and regenerative
medicine industry.
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