SEATTLE, Jan. 4, 2021 /PRNewswire/ -- AGC Biologics, a leading global Biopharmaceutical Contract Development and Manufacturing Organization (CDMO), is the first manufacturer of Orchard Therapeutics' Libmeldy, which was recently approved by the European Commission (EC) as a one-time therapy for eligible patients with early-onset Metachromatic Leukodystrophy (MLD).

The EC has granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy manufactured at AGC Biologics' Milan facility. Orchard Therapeutics is already underway with EU launch preparations to support commercial-scale drug manufacturing at the AGC Biologics Milan facility.

"AGC Biologics is delighted to have successfully partnered with Orchard Therapeutics on this great milestone," says AGC Biologics Chief Business Officer, Mark Womack. "We're very proud to be one of few Cell and Gene Therapy CDMOs with commercial experience Libmeldy being the third commercial product we've manufactured and we look forward to continuing our partnership with Orchard to provide this life-changing therapy to those affected by MLD."

"We are very honored to have been a part of the entire clinical journey of this product, from the very first treated patient, all the way to market approval. As manufacturer of both lentiviral vector and drug product, we are proud to support Orchard in treating this devastating disease," says AGC Biologics General Manager of Milan, Luca Alberici.

"The EC approval of Libmeldy opens up tremendous possibilities for eligible MLD children faced with this devastating disease," saysBobby Gaspar, M.D., Ph.D., Chief Executive Officer of Orchard. "We are humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU, and confident in the capabilities of our partners at AGC Biologics to help us achieve this goal."

MLD is a rare neurodegenerative disorder caused by mutations in the ARSA gene. While there are several forms of MLD, the disorder primarily affects young children and disease progression and life expectancy varies based on age of symptom onset. Libmeldy is designed to correct the genetic cause of MLD by inserting functional copies of the ARSA gene into the genome of a patient's own hematopoietic stem cells (HSCs) using a self-inactivating (SIN) lentiviral vector. It is approved in the EU for 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.

About AGC Biologics: AGC Biologics is a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO) with a strong commitment to deliver the highest standard of service as we work side-by-side with our clients and partners, every step of the way. We provide world-class development and manufacture of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), viral vectors and genetically engineered cells. Our global network spans the U.S., Europe and Asia, with cGMP-compliant facilities in Seattle, Washington; Boulder, Colorado; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba, Japan and we currently employ more than 1,600 employees worldwide. Our commitment to continuous innovation fosters the technical creativity to solve our clients' most complex challenges, including specialization in fast-track projects and rare diseases. AGC Biologics is the partner of choice. To learn more, visit http://www.agcbio.com.

About Orchard Therapeutics: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, please visit http://www.orchard-tx.com, and follow us onTwitterandLinkedIn.

About OTL-200/Libmeldy:Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union (as appropriate may add: "UK, Iceland, Liechtenstein and Norway" as an alternative can rewrite as "was approved by the European Commission" or something similar) 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 (AAA). 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).

Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy, which has not been approved by the U.S. Food and Drug Administration or any other health authority.

OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

SOURCE AGC Biologics

http://www.agcbio.com

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AGC Biologics Confirms Cell and Gene Therapy Commercial Expertise as Manufacturer of Orchard Therapeutics' Newly Approved Libmeldy - PRNewswire