Be The Match BioTherapies will provide
streamlined supply chain services in support of Orchard’s
commercial-stage gene therapy in Europe
Be The Match BioTherapies, an organization offering solutions
for companies developing and commercializing cell and gene
therapies, today announced an expansion of their multi-year
partnership with Orchard Therapeutics (Nasdaq: ORTX), a global gene
therapy leader, to include supply chain services in support of the
upcoming commercial launch of Libmeldy™ (autologous CD34+ cells
encoding the ARSA gene), Orchard’s gene therapy recently approved
in Europe for the treatment of early-onset metachromatic
leukodystrophy (MLD).
Through the expanded partnership, Be The Match BioTherapies will
provide comprehensive support across the commercial supply chain
for Libmeldy, including support of the onboarding and training of
apheresis centers, oversight of the autologous cell collection
process and delivery of both harvested cells to the manufacturing
site and gene-corrected cells back to the qualified treatment
center.
“Orchard’s mission to transform the lives of people living with
devastating genetic diseases like MLD is one that closely aligns
with our mission at Be The Match, which is to save lives through
cellular therapy,” said Chris McClain, Senior Vice President, Sales
and New Business Development at Be The Match BioTherapies.
“Leveraging our decades of experience and our far-reaching
capabilities across the cell therapy supply chain, including our
international network, we are well-positioned to support the
commercial launch of this important new gene therapy in
Europe.”
Be The Match BioTherapies previously supported cell collection
for Orchard’s clinical trials, and, through the expanded
partnership, will continue to enable streamlined logistical support
across each step of Libmeldy’s commercial development.
“HSC gene therapies are personalized medicines that require
precision to harvest a patient’s cells, transfer the cells to a lab
for genetic modification and then return the gene-corrected cells
back to a qualified treatment center to infuse into the patient,”
said Braden Parker, chief commercial officer of Orchard. “As we
move into the launch phase for Libmeldy in Europe, we are pleased
to continue our collaboration with Be The Match BioTherapies to
help enable us to maintain the efficient, high-quality supply chain
necessary to deliver Libmeldy to MLD patients in need.”
About Be The Match BioTherapies
Be The Match BioTherapies is the only cell and gene
therapy solutions provider with customizable services to support
the end-to-end cell therapy supply chain. Backed by the
industry-leading experience of the National Marrow Donor Program
(NMDP)®/Be The Match®, and a research partnership with the
CIBMTR® (Center for International Blood and Marrow
Transplant Research®), the organization designs solutions that
advance the development of cell and gene therapies across the
globe.
Be The Match BioTherapies is dedicated to accelerating patient
access to life-saving cell and gene therapies by providing
high-quality cellular source material from the Be The Match
Registry®, the world’s largest and most diverse registry of
more than 22 million potential blood stem cell donors. Through
established relationships with apheresis, marrow collection and
transplant centers worldwide, the organization develops, onboards,
trains and manages expansive collection networks to advance cell
therapies. Be The Match BioTherapies uses proven infrastructure
consisting of regulatory compliance and managed logistics experts,
as well as cell therapy supply chain case managers to successfully
transport and deliver regulatory compliant life-saving therapies
across the globe. Through the CIBMTR, Be The Match BioTherapies
extends services beyond the cell therapy supply chain to include
long-term follow-up tracking for the first two FDA-approved CAR-T
therapies.
For more information, visit
www.BeTheMatchBioTherapies.com or follow Be The Match
BioTherapies on LinkedIn or Twitter at
@BTMBioTherapies.
About MLD and Libmeldy/OTL-200
MLD is a rare and life-threatening inherited disease of the
body’s metabolic system occurring in approximately one in every
100,000 live births. MLD is caused by a mutation in the
arylsulfatase-A (ARSA) gene that results in the accumulation of
sulfatides in the brain and other areas of the body, including the
liver, gallbladder, kidneys, and/or spleen. Over time, the nervous
system is damaged, leading to neurological problems such as motor,
behavioral and cognitive regression, severe spasticity and
seizures. Patients with MLD gradually lose the ability to move,
talk, swallow, eat and see. In its late infantile form, mortality
at five years from onset is estimated at 50% and 44% at 10 years
for juvenile patients.1
Libmeldy (autologous CD34+ cell enriched population that
contains hematopoietic stem and progenitor cells (HSPC) transduced
ex vivo using a lentiviral vector encoding the human ARSA gene),
also known as OTL-200, has been approved by the European Commission
for the treatment of MLD in eligible early-onset patients
characterized by biallelic mutations in the ARSA gene leading to a
reduction of the ARSA enzymatic activity in children with i) late
infantile or early juvenile forms, without clinical manifestations
of the disease, or ii) the early juvenile form, with early clinical
manifestations of the disease, who still have the ability to walk
independently and before the onset of cognitive decline. Libmeldy
is the first therapy approved for eligible patients with
early-onset MLD.
The most common adverse reaction attributed to treatment with
Libmeldy was the occurrence of anti-ARSA antibodies. In addition to
the risks associated with the gene therapy, treatment with Libmeldy
is preceded by other medical interventions, namely bone marrow
harvest or peripheral blood mobilization and apheresis, followed by
myeloablative conditioning, which carry their own risks. During the
clinical studies, the safety profiles of these interventions were
consistent with their known safety and tolerability.
For more information about Libmeldy, please see the Summary of
Product Characteristics (SmPC) available on the European Medicines
Agency (EMA) website.
Libmeldy is not approved outside of the European Union, UK,
Iceland, Liechtenstein, and Norway. OTL-200 is an investigational
therapy in the U.S.
Libmeldy was developed in partnership with the San Raffaele
Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
About Orchard
Orchard Therapeutics is a global gene therapy leader dedicated
to transforming the lives of people affected by rare diseases
through the development of innovative, potentially curative gene
therapies. Our ex vivo autologous gene therapy approach harnesses
the power of genetically modified blood stem cells and seeks to
correct the underlying cause of disease in a single administration.
In 2018, Orchard acquired GSK’s rare disease gene therapy
portfolio, which originated from a pioneering collaboration between
GSK and the San Raffaele Telethon Institute for Gene Therapy in
Milan, Italy. Orchard now has one of the deepest and most advanced
gene therapy product candidate pipelines in the industry spanning
multiple therapeutic areas where the disease burden on children,
families and caregivers is immense and current treatment options
are limited or do not exist.
Orchard has its global headquarters in London and U.S.
headquarters in Boston. For more information, visit
www.orchard-tx.com, or follow Orchard on Twitter and LinkedIn.
Forward-Looking Statements
This press release contains certain forward-looking statements
which are made pursuant to the safe harbor provisions of the
Private Securities Litigation Reform Act of 1995. Such
forward-looking statements may be identified by words such as
“anticipates,” “believes,” “expects,” “intends,” “projects,”
“anticipates,” and “future” or similar expressions that are
intended to identify forward-looking statements. Forward-looking
statements include express or implied statements relating to, among
other things, Orchard’s business strategy and goals, including its
plans and expectations for the commercialization of Libmeldy
(OTL-200) in Europe, the therapeutic potential of Libmeldy and
Orchard’s product candidates, and the expected benefits from
Orchard’s partnership with Be The Match BioTherapies. These
statements are neither promises nor guarantees, but are subject to
a variety of risks and uncertainties, many of which are beyond
Orchard’s control, which could cause actual results to differ
materially from those contemplated in these forward-looking
statements. These risks and uncertainties include, without
limitation: risks relating to the Company’s inability, or the
inability of Be The Match BioTherapies, to support a successful
commercial launch of Libmeldy. Orchard undertakes no obligation to
publicly update or revise any forward-looking statements, whether
as a result of new information, future events or otherwise, except
as may be required by law.
Other risks and uncertainties faced by Orchard include those
identified under the heading "Risk Factors" in Orchard’s annual
report on Form 10-K for the year ended December 31, 2020, as filed
with the U.S. Securities and Exchange Commission (SEC), as well as
subsequent filings and reports filed with the SEC. The
forward-looking statements contained in this press release reflect
Orchard’s views as of the date hereof, and Orchard does not assume
and specifically disclaims any obligation to publicly update or
revise any forward-looking statements, whether as a result of new
information, future events or otherwise, except as may be required
by law.
1 Mahmood et al. Metachromatic Leukodystrophy: A Case of
Triplets with the Late Infantile Variant and a Systematic Review of
the Literature. Journal of Child Neurology 2010, DOI:
http://doi.org/10.1177/0883073809341669
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version on businesswire.com: https://www.businesswire.com/news/home/20210303005209/en/
Be The Match BioTherapies: Gwen Schanker Ten Bridge
Communications (269)-921-3607 gwen@tenbridgecommunications.com
Orchard Therapeutics: Media: Christine Harrison Vice
President, Corporate Affairs +1 202-415-0137 media@orchard-tx.com
Investors: Renee Leck Director, Investor Relations +1
862-242-0764 Renee.Leck@orchard-tx.com
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