Cellectis ($CLLS) has completed three production runs of its off-the-shelf CAR-T therapy under GMP conditions. The news sent shares in Cellectis up by 20% in early trading in Paris, emphasizing the key role manufacturing know-how is seen as playing in the upcoming immuno-oncology scrap.
Paris, France-based Cellectis has stepped up the development of its manufacturing plans this year, bringing Arjan Roozen on board to oversee the operation in March and presenting a poster at ASCO several months later. The drive has brought the gene-editing specialist to the point at which it can produce its Servier-partnered lead candidate UCART19 under regulatory-grade conditions. While the big questions about the safety and efficacy of the drug are yet to be answered, the production runs cut the risk of Cellectis' allogeneic CAR-T therapies being impeded by manufacturing issues.
Cellectis' focus on off-the-shelf products is one of the ways in which it differentiates itself from its rivals for the immuno-oncology crown. "Historically, cell-based therapies have grown in the world of individual grafts," Cellectis EVP David Sourdive said in a statement. "With Talen-based gene editing they have now started moving toward that of industrial pharmaceutical products broadly available to patients." The industrialization of manufacturing is key to Cellectis' ambition to achieve gross margins of up to 90%. Dendreon, with its personalized immunotherapy, never got close to this level.
The first generation of CAR-T therapies coming down the pipeline at Novartis ($NVS), Kite Pharma ($KITE) and Juno Therapeutics ($JUNO) are produced by manipulating patients' T cells, a process that Cellectis CEO André Choulika sees as condemning them to niche status. Each of the firms in the first wave of CAR-T has taken steps to stop manufacturing from contributing to the realization of Choulika's forecast. Novartis picked up a plant from Dendreon for $43 million (€39 million) in 2012, while Juno and Kite made near-simultaneous investments in in-house capacity earlier this year.
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