Orchard Therapeutics says it will balance its existing CMO network with a planned facility in Fremont to create the infrastructure needed to commercialize its gene therapies.

Orchard’s pipeline of ex vivo autologous gene therapies include the former GSK hematopoietic stem cell (HSC) gene therapy Strimvelis – approved by the EMA in 2016 – and five clinical stage programs.

The firm has been reliant on its network of contract manufacturing organizations (CMOs) to produce these therapies, but late last year it announced it had signed a long-term lease agreement to build-out a gene therapy manufacturing facility in Fremont, California to support manufacturing of lentiviral vector and cryopreserved cell therapy products.

Image: AdobeStock/Aleksandar Mijatovic

On a conference call to discuss its most recent quarter, chief scientific officer Bobby Gaspar confirmed the design work is “well under way” and the firm remains on track to open the 150,000 square-foot plant in 2021.

Chief technical officer, Ran Zheng, gave Bioprocess Insider more information on the rationale for Orchard’s expansion:

“The diseases we target affect patients around the world, which require us to have the infrastructure to deliver gene therapies globally. We are therefore building a commercial-scale manufacturing infrastructure and leveraging technologies that will allow us to deliver our gene therapies globally and in a fully-integrated manner.”

She added that in order to meet anticipated demand for its growing pipeline of product candidates, the firm is initially using CMOs.

For its ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency) treatments, Oxford BioMedica handles vector manufacturing and Lonza handles the drug product manufacturing. For its candidates looking to treat metachromatic leukodystrophy (MLD) and Wiskott–Aldrich syndrome (WAS), manufacturing operations are currently being managed by development partner MolMed S.p.A, following an agreement inked between the two firms in December 2018.

While Orchard looks to Fremont for manufacturing, the firm has established some inhouse functions, including process development capabilities at its Menlo Park, California and London, UK sites.

“We are also developing and implementing cryopreservation processes for our clinical stage product candidates, which, in combination with our international network of CMOs and our planned in-house manufacturing capabilities, will help enable the distribution and administration of our gene therapies to wherever patients are located across the globe.”