Be The Match BioTherapies® Announces Expansion of Multi-Year Strategic Alliance with Orchard Therapeutics to Support European Commercial Launch of Libmeldy™

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Be The Match BioTherapies will provide streamlined supply chain services in support of Orchard’s commercial-stage gene therapy in Europe

MINNEAPOLIS, Minn., March 3, 2021 – 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.”


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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

Be The Match BioTherapies Media Contact:
Gwen Schanker
Ten Bridge Communications
(269)-921-3607
gwen@tenbridgecommunications.com

Orchard Therapeutics Contacts:
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