Orchard picks Yposkesi to make vectors for lentiviral gene therapies

By Flora Southey

- Last updated on GMT


Related tags Immune system Gene therapy

Yposkesi will make lentiviral vectors for Orchard Therapeutics’ gene therapies – indicated to treat primary immunodeficiencies – at its facility near Paris, France.

Under the agreement, Yposkesi will use its own platform to make vector batches at its Corbeil-Essonnes facility for Orchard’s clinical trials.

“The production process is based on cell culture using the HEK293 cell line, quadruple transfection with plasmids, followed by purification and aseptic filling,” ​a Yposkesi spokesperson told us.

The contract manufacturing organisation (CMO) said it will not be investing in additional technology for the project: “We have all the facilities and equipment required.”

Autologous ex-vivo ​lentiviral gene therapy – which uses lentiviruses to modify or delete genes – is a potential treatment modality for a significant number of illnesses, Orchard CEO Mark Rotherea told us.

Potential indications include primary immune deficiencies such as adenosine deaminase severe combined immunodeficiency (ADA-SCID) and X-linked chronic granulomatous disorders (X-CGD), as well as inherited metabolic disorders such as mucopolysaccharidosis type IIIA and IIIB, and blood disorders such as thalassemia and sickle cell disease, he said.

Orchard CEO Mark Rotherea told us the partnership is part of a wider alliance with Yposkesi’s parent company Genethon.

“Yposkesi has developed the capabilities of viral vector and cell product manufacture and has successfully manufactured lentivirus for trials sponsored by Genethon and the University of California, Los Angeles (UCLA) to support clinical studies in X-CGD in Europe and the United States,” ​he told us.

“X-CGD will be the first indication in which Orchard and Yposkesi are collaborating,” ​he added. 

Clinical stage

Orchard is partnering with transplant centres to perform clinical studies, Rotherea told us.

“To conduct clinical trials with autologous ​ex-vivo lentiviral gene therapy, Orchard is collaborating with transplant centres specialised in the treatment of primary immune deficiencies, inherited metabolic disorders, and blood disorders,” ​he said.

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