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Gilead Sciences has claimed another stake in the emerging cell therapy sector with a $3bn agreement to use Sangamo Biosciences gene-editing platform for new cell-based cancer therapies.
The company seems determined to stay in the forefront of the pharma industry’s push into cell therapies, with the latest deal coming shortly after it acquired CAR-T specialist Kite Pharma for $12bn and almost immediately bolted on another CAR-T tech through the takeover of Cell Design a few weeks later.
Sangamo is getting $150m upfront from Gilead in the deal, which will see Kite claim an exclusive license to use the biotech’s zinc finger nuclease (ZFN) technology to develop up to ten off-the-shelf (allogeneic) as well as autologous CAR-T therapies. It also stands to receive up to $3bn in milestones – $300m per product – as well as tiered royalties on sales.
Kite will be responsible for all development, manufacturing and commercialisation of products under the collaboration, and will be responsible for agreed upon expenses incurred by Sangamo.
Gene-editing techniques are used to modify the cells – either harvested from patients or taken from donor stocks – that are infused into cancer patients in order to mount an immunotherapeutic assault on cancers. The deal with Sangamo means rivals in the CAR-T category such as Novartis, Celgene/Juno and off-the-shelf specialist Cellectis won’t be able to use the ZFN technology in their own gene-editing toolboxes.
ZFN is a gene-editing approach that uses a DNA-cutting nuclease enzyme attached to zinc finger -binding proteins to recognize and edit specific sequences of DNA. Other techniques include CRISPR/Cas9 – used by Novartis and Juno – and Cellectis’ favoured TALENS.
However, according to CEO Sandy Macrae, Sangamo has made significant strides in improving the precision, efficiency and specificity of ZFN, which means the technique now “sets the standard on what therapeutic genome editing should be”.
He said that Kite’s “financial strength and clear determination” to bring new cellular therapies products forward makes it an ideal partner for ZFN in this setting.
Kite highlighted the potential of the technology for developing allogeneic CAR-Ts, which have been put forward as potentially a major advance over autologous therapies, as they should be much quicker and cheaper to deliver – an important consideration given the current debate over the escalating costs of cancer treatment.
“The emergence of gene editing as a tool to edit immune cells holds promise in the development of therapies with potentially improved safety, efficacy and efficiency,” commented Gilead’s CEO John Milligan.