Kite Gains Access to Refuge's Gene Expression Platform in Licensing Deal

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Gilead Sciences' Kite Pharma entered into a global licensing deal with Refuge Biotechnologies Thursday to leverage the latter's propriety gene expression platform and develop potential blood cancer therapies.

Under the terms of the deal, Kite has exclusive access to Refuge's intellectual property portfolio for use in blood cancers. It also has an exclusive license to Refuge's library of synthetic gene expression programs for those hematological indications.

Refuge keeps all rights and programs associated with solid tumors.

Through this license deal with Refuge's platform, the company aims "to create a new generation of CAR T-cell products to induce long-term remissions for more patients," Francesco Marincola, MD, global head of cell therapy research for Kite, stated.

"First-generation autologous CAR T-cell therapies have dramatically changed outcomes for people with certain blood cancers, yet more work needs to be done to reach additional patients," Marincola added.

Refuge's technology platform is a synthetic biology system. Its strategy involves expression modulation to repress or activate the transcription of target genes.

Early preclinical research points the way to the platform to regulate target antigen-dependent gene expression to improve the efficacy and safety of first-generation CAR-T therapies. 

CAR-T has been successfully developed for blood cancers, but its effectiveness in solid tumors remains elusive.

"One of the main limitations is that most of the proteins present on solid tumors that could be used as targets are also found at low levels on normal cells, making it difficult to specifically direct the CAR T cells against tumor cells and spare healthy ones," John Haanen, MD, Ph.D., a medical oncologist at the Netherlands Cancer Institute, said during a presentation at the American Association for Cancer Research meeting in April.

"Other challenges include the limited persistence of CAR T cells observed in solid tumors and their difficulty reaching the tumors and penetrating the center of the mass," Haanen added.

Kite will handle all costs and activities associated with research, development, manufacturing and commercialization. The company will also pay Refuge an undisclosed upfront payment. Refuge is also eligible for various performance-based regulatory milestones.

The FDA has approved six CAR T-cell therapies since 2017. One of them is Kite's Yescarta for five types of blood cancers. Another is Tecartus for adults with relapsed or refractory mantle cell lymphoma and adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

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