Third Rock-backed startup launches to develop cell therapies for MS, diabetes

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Five cancer cell therapies are approved in the US and others are in clinical testing as drug makers work to reuse human cells as platforms for new drugs, many of which are similarly targeted at different cancers.

But more and more biotechnology companies are aiming to use cell therapy for diseases that turn the body’s immune system against itself.

“We are in the era of building cells to fulfill our commandments,” said Samantha Singer, the CEO of a new startup that launches Wednesday with $ 95 million in venture capital funding. “We should do this with autoimmune diseases.”

For the past three years, Singer and scientists from Third Rock Ventures, the company’s principal financier, Harvard Medical School, the Massachusetts Institute of Technology, and the National Institutes of Health have explored this idea leading to the development of the newly launched company.

The startup called Abata Therapeutics plans to genetically develop 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 leading MS therapy by 2024 and to begin clinical trials of treatments for the other two diseases by the end of 2025.

“Now is the time for Abata because technology has advanced so far that we can actually do some of the things we dreamed of two or three years ago,” said Diane Mathis, professor of immunology at Harvard Medical School and a co-founder of Abata, in a video announcing the company’s launch.

The cells that Abata works with, the so-called regulatory T cells or Tregs, are a central component of the body’s immune system. It is crucial that they also have many of the properties that Singer and the Abata team were looking for in therapy.

“The ideal therapy would be something that works locally, only in tissue with an autoimmune disease,” Singer said in an interview. “You want something that counteracts multiple inflammatory mechanisms and promotes repair. You want something that will last. That’s what a Treg does in your immune system. “

Abata plans to engineer these Tregs using a cellular receptor specifically designed to recognize antigens – protein flags that act as triggers for an immune response – that are associated with inflamed tissue.

“I like to think that we just use that strong, wise immune system that nature has given us,” said Richard Ransohoff, a neurologist and partner at Third Rock, who co-founded Abata, in an interview.

Abata will initially target a progressive form of MS, a disease in which the immune system attacks the protective covering of nerve cells. There are many drugs that are now approved for relapsing forms of MS, and one, Roche’s Ocrevus, is approved for the primary progressive stage of the disease.

But for patients with progressive MS who no longer have active relapses, Abata says there is no treatment option.

“NoIn the ribs of the brain and spinal space, the cells of immune-active cells have lodged, “said Ransohoff, who described the brain as a walnut, with alleys that can accommodate these nodules.

Ocrelizumab does not touch these inflammatory nodules that live in the central nervous system and will stay there after all peripheral inflammation is removed, ”he added, using the scientific name of Ocrevus.

Abata estimates that there are approximately 45,000 patients in the United States with progressive MS with non-relapsing disease and persistent inflammatory tissue damage. The company plans to identify these patients using an MRI biomarker developed by NIH researcher Daniel Reich, a scientific advisor to Abata.

In addition to Third Rock, Abata is also supported by ElevateBio, a richly funded and unusual biotech company that combines in-house research with a partner company to help other companies manufacture complex cell therapies.

Lightspeed Venture Partners, Invus, Samsara BioCapital and the JDRF T1D Fund also invested in Abata as part of the Series A funding round led by Third Rock and ElevateBio.

Abata isn’t the only one trying to build cell therapies around Tregs, but it appears to be one of the best-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.