Five cancer cell therapies are approved in the U.S. Scores more are in clinical testing as drugmakers work to repurpose human cells as a platform for new medicines, many of which are similarly targeted at different types of cancer.
But more and more biotechnology companies aim to use cell therapy for diseases that turn the body's immune system against itself.
"We're in the era of engineering cells to do our bidding," said Samantha Singer, the CEO of a new startup launching Wednesday with $95 million in venture capital funding. "We should do this in autoimmune disease."
Over the past three years, Singer and scientists from Third Rock Ventures, the company's principal venture backer, Harvard Medical School, Massachusetts Institute of Technology and the National Institutes of Health have been studying that idea, work that led to the creation of the newly debuted company.
Called Abata Therapeutics, the startup plans to genetically engineer a type of regulatory immune cell to treat multiple sclerosis, Type 1 diabetes and an inflammatory disease called inclusion body myositis. If all goes well, Abata expects to have early clinical trial data on its lead therapy for MS by 2024 and to start clinical testing of treatments for the other two diseases by the end of 2025.
"Now is the time for Abata because the technology has evolved to a point where we can actually do some of the things that we were dreaming of two or three years ago," said Diane Mathis, a professor of immunology at Harvard Medical School and a co-founder of Abata, in a video announcing the company's launch.
The cells Abata chose to work with, called regulatory T cells, or Tregs, are a core part of the body's immune system. Crucially, they also possess many of the attributes Singer and the Abata team were looking for in a therapy.
"The ideal therapy would be something that acts locally, only in tissue that has an autoimmune pathology," said Singer, in an interview. "You'd want something that could counter multiple mechanisms of inflammation and promote repair. You'd want something that's durable. That's what a Treg does in your immune system."
Abata plans to engineer these Tregs with a cell receptor specifically designed to recognize antigens — protein flags that act as triggers for an immune response — associated with inflamed tissue.
"I like to think that we are simply taking advantage of this powerful, wise immune system that nature gave us," said Richard Ransohoff, a neurologist and partner at Third Rock who co-founded Abata, in an interview.
Abata plans to first target a progressive form of MS, a disease in which the immune system attacks the protective covering of nerve cells. There are many drugs now approved for relapsing forms of MS and one, Roche's Ocrevus, cleared for the primary progressive stage of the disease.
But for patients with progressive MS who no longer experience active relapses, there's no treatment option, according to Abata.
"Nodules of immune active cells have become lodged in corrugations of the brain and spinal space," said Ransohoff, who described the brain as a walnut, with alleys that can harbor those nodules.
"Ocrelizumab doesn't touch these nodules of inflammation that live in the central nervous system and are going to stay there after all the peripheral inflammation is removed," he added, using Ocrevus' scientific name.
Abata estimates there are about 45,000 patients in the U.S. who have progressive MS with non-relapsing disease and ongoing inflammatory tissue injury. The company plans to identify these patients using an MRI biomarker developed by NIH researcher Daniel Reich, who is a scientific adviser for Abata.
Along with Third Rock, Abata is also backed by ElevateBio, a richly funded and unusual biotech that mixes in-house research with a partnering business helping other companies manufacture complex cell therapies.
Lightspeed Venture Partners, Invus, Samsara BioCapital and the JDRF T1D Fund also invested in Abata through the Series A funding round led by Third Rock and ElevateBio.
Abata is not alone in attempting to build cell therapies around Tregs, but it appears to be one of the most well-funded. GentiBio, Quell Therapeutics, Sonoma Biotherapeutics, Coya Therapeutics and, most recently, TRexBio, have all launched in recent years with plans to use Tregs to treat autoimmune diseases.