Kite, a Gilead Company, will use Sangamo Therapeutics’ zinc finger nuclease (ZFN) gene-editing technology platform in a global oncology cell therapy collaboration that could generate up to $3.16 billion-plus for Sangamo, the companies said today.

Kite plans to modify genes through Sangamo's ZFN technology in order to develop autologous and allogeneic next-generation cell therapies for different cancers. Those cell therapies would be “ex vivo”; cells would be removed from the body, modified, and then then infused back in.

The companies reason that whether from healthy donor cells or from renewable stem cells, allogeneic cell therapies would provide a potential treatment option that can be accessed directly within the oncology infusion center, thus reducing the time to infusion for patients.

Sangamo has been studying ZFNs for 20 years and is the primary patent holder for the technology. “What we know is the ZFN technology works. There’s no question about that,” Charles Gersbach, Ph.D., Rooney Family Associate Professor of Biomedical Engineering at Duke University, told GEN late last year.  

Through years of R&D efforts by Sangamo, ZFNs have achieved several firsts in the gene-editing space: the first to edit genes in human cells, the first to edit genes ex vivo—and the first to edit genes in vivo (i.e., editing genes in cells still intact in the body).

On November 15, Sangamo disclosed that it edited in vivo the genes of the first patient in the Phase I/II CHAMPIONS trial to treat a rare genetic disorder. The patient's veins were infused with Sangamo’s investigational genome-editing therapy SB-913 to treat his disease, mucopolysaccharidosis type II (MPS II). MPS II, often called Hunter syndrome, is caused by a mutation in the iduronate 2-sulfatase (IDS) gene and occurs almost exclusively among males. The therapy uses ZFN to cut out the IDS mutated gene and replace it with the healthy gene.

CAR-T Scramble

“We believe Sangamo's ZFNs provide the optimal gene-editing platform, and we look forward to working with Sangamo to accelerate our efforts to develop next-generation autologous cell therapies, as well as allogeneic treatments that can be accessed more conveniently in the hospital setting for people living with cancer,” Gilead Sciences president and CEO John F. Milligan, Ph.D., said in a statement.

Gilead catapulted itself to a leading position in the scramble to develop chimeric antigen receptor T-cell (CAR-T) cancer therapies last year when it acquired Kite Pharma for $11.9 billion, in a deal completed October 3. Fifteen days later, Kite won the FDA’s second approval of a CAR-T treatment for a form of cancer when the agency authorized Yescarta™ (axicabtagene ciloleucel). Yescarta is the first CAR-T therapy indicated for adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma (transformed follicular lymphoma).

Gilead signaled it intended to grow its footprint in next-generation CAR-T treatments as well in December, when it agreed to acquire Cell Design Labs for up to approximately $567 million.

Also competing in next-gen CAR-T are Celgene, which last month agreed to acquire Juno Therapeutics for approximately $9 billion; and Novartis, which won the FDA’s first CAR-T therapy approval for Kymriah™ (tisagenlecleucel) in August, and has CRISPR-based gene-editing collaborations with Intellia Therapeutics and Caribou Biosciences launched in 2015

In the latest collaboration, Kite has agreed to pay Sangamo $150 million upfront, and up to $3.01 billion in payments tied to achieving research, development, regulatory, and successful commercialization milestones. The payments will be aggregated across 10 or more products using Sangamo's technology.

Sangamo would also be eligible to receive tiered royalties from Kite on sales of potential future products resulting from the collaboration.

“Two Leading Platforms”

Kite will oversee all development, manufacturing, and commercialization of products under the collaboration, and has agreed to shoulder agreed-upon expenses incurred by Sangamo, the companies said.

“This collaboration between Kite and Sangamo brings together two leading platforms to develop best-in-class cell therapies in oncology,” added Sangamo president and CEO Sandy Macrae, Ph.D. “We are excited by Kite's commitment to driving innovation in this field and look forward to working together to realize the full promise of cell therapy in treating cancer.”

The collaboration is the second $3 billion-plus oncology partnership announced in as many weeks, and Sangamo’s second partnership with a biopharma giant in as many months.

On February 14, Bristol-Myers Squibb and Nektar Therapeutics launched a cancer immunotherapy alliance that could yield up to $3.6 billion-plus for Nektar.

Last month, Sangamo and Pfizer began an up-to-$162 million-plus alliance to develop a potential gene therapy to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene—the companies’ second gene therapy collaboration.

The Kite-Sangamo collaboration is subject to clearance under the Hart–Scott–Rodino Antitrust Improvements Act and other customary closing conditions, Kite and Sangamo added.

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