Gilead’s CSO spoke about its choice of zinc finger nuclease (ZFN) for ex-vivo gene editing at the Bank of America Merrill Lynch Healthcare Conference.
In February, Kite Pharma – acquired by Gilead Sciences for US$11.9 billion in last August – struck a deal to use Sangamo Therapeutic’s ZFN technology to modify genes in the development of its autologous and allogeneic cell therapies.
Kite/Gilead paid $150 million up front to the fellow Californian firm but Sangamo could be entitled to a further $3 billion in milestone payments and royalties from upwards of 10 products being developed using its ZFN tech.
Zinc-finger nucleases are enzymes that allow scientists to edit the genome of a mammalian cell, knocking out unwanted genes in a precisely targeted way.
Speaking at the Bank of America Merrill Lynch Healthcare Conference last week, Gilead’s CSO and head of R&D John McHutchison explained why his firm selected ZFN over other emerging technologies being used by cell and gene therapy developers: transcription activator-like effector nucleases (TALENs) and CRISPR-Cas9.
“We did obviously a lot of diligences on all the different technologies and in terms of what we needed for ex-vivo gene editing for autologous and off the shelf allogeneic cells, cell manipulation,” he told delegates (transcript here).
He described the tech as an efficient, precise, and specific approach to gene editing, and will be used to in the firm’s autologous and off-the-shelf pipeline.
“It’s an integral part of being able to move forward with second generation product gene editing for off-the-shelf products, potentially also for the solid tumor initiatives as well, and cell design.”
Kite is one of two firms to have achieved regulatory success with an autologous chimeric antigen receptor (CAR) T cell therapy. Yescarta (axicabtagene ciloleucel) received US Food and Drug Administration (FDA) approval to treat adults with large B-cell lymphoma in October last year, weeks after Novartis received the thumbs up for its CAR-T cell therapy Kymriah (tisagenlecleucel).