As CEO of Rubius Therapeutics, Pablo Cagnoni oversaw the development of a cell therapy made from red blood cells, but was at Novartis when the pharma giant struck a deal with T-cell research, which is proving to be an area of cell therapy The Beacon. CAR T therapy offers a new way to treat some of the toughest blood cancer cases, but Cagnoni also acknowledges the limitations of the therapy.
These personalized therapies made from a patient’s own T cells are not scalable. Despite all the therapeutic benefits, CAR T still poses dangerous safety risks to patients. Furthermore, the effectiveness of this treatment against liquid tumors has proven elusive in solid tumors. Now, 10 years after Novartis licensed the University of Pennsylvania’s CAR T technology, which will become Kymriah, and five years after the cell therapy’s landmark FDA approval, Cagnoni says he expects the field to progress more than he expected. slow.
“These three barriers remain, at least for CAR T cells,” Cagnoni said.
Canioni spoke Monday during a panel discussion on the potential of cell and gene therapy. World Medical Innovation Forum in Boston. While the panelists acknowledged these challenges, they also pointed to the approaches that cell and gene therapy research is taking to overcome them.
Bristol-Myers Squibb is working to improve CAR T in several different ways, said Kristen Hege, senior vice president of early clinical development, hematology/oncology and cell therapy at Bristol-Myers Squibb. The company is optimizing autologous cell therapy by automating the process and improving how these cells are designed. The next-generation cell therapy being developed by BMS uses gene-editing techniques to knock out parts of the genome that could allow cells to mount a dangerous immune response, or to knock in properties you want to make as a treatment more effective, Hege said. Many editors build on what has been learned from first-generation CAR T therapies.
Cell therapy research is going beyond T cells.notice Nktarta Therapeutics reported encouraging early clinical data for its engineered and off-the-shelf natural killer cells last week, Cagnoni said, other types of cells may be able to overcome some of the challenges facing T-cell-based therapies. Andrew Plump, Takeda’s president of research and development, said his company’s cell therapy work focuses on innate immune cells. Takeda doesn’t do as much gene-editing research as other companies. But Plump said that, similar to BMS, Takeda’s approach uses editing to improve certain properties of cell therapy, such as ensuring delivery to the right tissues.
Chroma Medicine CEO Catherine Stehman-Breen said cell and gene therapies are opening the door to more complex diseases through treatment. These therapies also have the potential to address a broader patient population, she added. Her Cambridge, Massachusetts-based startup is developing epigenetic drugs that treat disease by turning genes on or off. Chroma launched last fall with a $125 million Series A roundChroma’s approach builds on earlier work in cell and gene therapy research, Stehman-Breen said.
Takeda’s approach includes partnering with smaller biotech companies like Chroma to gain capabilities it doesn’t have in-house, Plump said. Takeda’s partnership with bispecific T cell engagement developer Maverick Therapeutics led to the biotech’s acquisition last year. Plump said the deal is the product of an ongoing relationship between the two companies.
Brisbane, California-based Maverick has the platform technology to make bispecific drugs, but it doesn’t have the capability to make cell lines. Takeda provides these capabilities, leading the two companies to work more closely together. The partnership includes an equity investment, giving the pharma giant the option to acquire Maverick. Innovation in those things that biotechs don’t do well within Takeda will continue to drive the company’s investments. Plump offered a prediction, saying, “We think that within seven to 10 years, 50 percent of our clinical-stage pipeline will be in cell and gene therapy.”
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