Gene editing plays a major role in next-gen CAR-T programs. And Cellectis can now boast of a patent that touches on a broad range of technologies that will come into play.
The U.S. patent office has issued patent number 9,458,439, protecting the work that Cellectis CEO (and scientist) André Choulika and Harvard Professor Richard C. Mulligan, one of the original pioneers in the gene therapy field, have accomplished.
Put simply, the patent covers a variety of technologies they used to perform surgery on a gene in vitro.
‘This patent doesn’t focus on one technology,” Choulika tells me this morning in a call from Paris, where Cellectis is based. Rather, it encompasses several ways to induce gene inactivation, citing roles for CRISPR/Cas9, Zinc finger Nucleases, TAL-Effector Nucleases, Mega-TALEs and some Meganucleases.
“It’s a large way to see gene editing. This, for me, is the basis for nuclease-based gene editing,” he adds. “It positions us in a very competitive way to move forward in the space.”
Choulika and his partners at Pfizer and Servier are now in early-stage testing for an allogeneic approach to CAR-T, using gene editing technologies to engineer an off-the-shelf cell therapy that would radically change the landscape now dominated by late-stage efforts on personalized therapies at Kite, Juno and Novartis that extract cells and then reengineer them into cancer treatments.
Don’t expect gene-editing tech to remain unchanged, adds Choulika, who’s Beirut-born, with a PhD from the University of Paris and research stints in genetics at Harvard and Boston Children’s behind him.
“The cell phone you’re holding in your hand is obsolete,” he says by way of comparison. And so is the gene editing tech you’re seeing at the forefront of the arena now. “If you marry yourself to a technology, you’re doomed.”
This is also a fast-moving development. In just recent weeks Novartis $NVS has dissolved it’s 400-member cell therapy unit. Juno $JUNO, allied with Editas on gene editing, has been dealing with the fallout of a brief clinical hold on its lead therapy, which derailed its approval schedule. That leaves Kite $KITE out front with plans to hustle a new drug application to the FDA after only three months of results to work with.
“I’m very impressed by what Kite has been doing,” says Choulika. Their autologous R&D work has shown that there is a clear path to the market, and that will help demonstrate that there’s a path for Cellectis as well.
Says the CEO: “Kite is definitely leading the way.”
And Cellectis is moving as fast as it can to follow up.
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