Refuge Biotechnologies has announced that it has raised $25 million in a round of Series B financing, money which will be used to develop its pipeline of intelligent cell therapies.

The company also announced the appointment of immuno-oncologist Francesco Marincola as chief scientific officer.

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Refuge uses of gene engineering technologies to develop intelligent cell therapeutics designed to make decisions inside patients’ bodies. Its receptor-dCas platform uses mutated or dead Cas9 (dCas) as a targeting mechanism, which enables precision CRISPR activation (CRISPRa) and CRISPR interference (CRISPRi).

The dCas9 protein does not cut DNA but rather acts as a carrier to particular areas of the genome, enabling more precise targeted delivery. The platform offers control over a cell’s interaction with its environment as it enables cells to sense their surroundings and activate or repress multiple gene expressions based on the receptor-ligand interactions. Receptor-dCas can programme cells to inhibit certain genes and generate more potent CAR-T immune cells when it senses cancerous cells.

The cell therapies are programmed to activate CRISPRa/CRISPRi only when they come into contact with particular sensors on the surface of cancer cells, allowing the treatment to target specific cells.

This method allows multiple cancer treatments to be combined within a single cell, enabling greater potency and reduced side effects. Refuge’s lead candidate is RB-1916, a CAR-T cell therapy designed to hinder the gene PD-1, which plays a key role in down-regulating the immune system. RB-1916 has a potential initial application in diffused large B-cell lymphoma.

“We have seen tremendous progress in the development of our technology and science, and believe that our receptor-dCas platform has the potential to create highly targeted cell therapies that bring superior efficacy while overcoming limitations related to toxic side effects,” Refuge CEO Dr Bing C. Wang said.

“This financing will propel our efforts with our growing pipeline as we continue to design these innovative and intelligent cell therapies to fight cancer, and we are encouraged by the support from this top group of global investors.”

The financing round was led by 3SBio and Sequoia China, with new investors including Danhua Capital (DHVC), Sangel Capital and Ocean Pine Healthcare Fund.

Under the terms of the investment, the lead investors will have exclusive rights to negotiate with Refuge on the development and commercialisation of cell therapies using Refuge’s platform in China. Additionally, Refuge and 3SBio are to collaborate on research into programmed cell therapies, which produce therapeutic proteins inside a patient’s body using Refuge’s platform.

3SBio’s investment demonstrates our commitment to  advancing cutting-edge gene engineering technologies with potential for breakthrough treatments for cancer and other diseases with unmet medical needs,” 3SBio CEO Jing Lou said.

“3SBio looks forward to collaborating with Refuge to accelerate the clinical development of Refuge’s next-generation cell therapies for cancer and to fully realise the potential of the dCas9 platform.”