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Fresh Or Frozen? Orchard Aims For Cryopreserved Gene Therapy Approvals Using Pivotal Trials With Fresh Cell Formulations

 
• By Bridget Silverman

Orchard aims to submit three ex vivo autologous gene therapies for approval by the end of 2021, helped by a new regenerative medicine advanced therapy designation (for a rare primary immunodeficiency syndrome) and a breakthrough therapy designation (for another rare primary immunodeficiency).

Orchard Therapeutics Ltd.'s ambitious plans to submit three gene therapy candidates for US and European approval in 2020 and 2021 assumes that the company can meet the test of any application – demonstrating safety and efficacy – while also showing that the cryopreserved gene therapies to be produced commercially by Orchard and its contractors is the same as the fresh cells produced by clinical investigators in the registration trials.

The challenge facing Orchard is inherent in the transition of autologous gene therapy from what was essentially a bespoke model of individualized therapies brewed up at academic medical centers to a commodity model

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