Startups, Venture

Penn spinout preps for human studies of a new-model CAAR, aiming at a one-time cure for autoimmune diseases

Michael Milone

Michael Milone learned about engineering T cells for cancer at the bench of the master, working as a post doc in Carl June’s lab at the University of Pennsylvania from 2004 to 2007. He went on to play a key role in the scientific leadership team developing Novartis’ breakthrough CAR-T drug Kymriah at Penn. And now, at the head of his own team of translational researchers, he and Penn’s Aimee Payne and Steven Nichtberger are co-founding a biotech that will take what they’ve learned about T cell engineering into autoimmune diseases, in search of a breakthrough, once-and-done approach.

The upstart — Cabaletta Bio, to be helmed by Nichtberger — has grabbed a $38 million round from a group of investors with an eye to going into the clinic with the work they’ve done on mucosal pemphigus vulgaris, a rare skin blistering disease, recently published in Science.

Aimee Payne

So what’s the big idea here? The team at Penn believes they have constructed a next-gen CAAR — with Chimeric AutoAntibody Receptors. These CAARs have shown a preclinical ability to identify and eliminate the specific B cells that mediate a wide variety of autoimmune conditions, while leaving the normal B cells alone. And they’re betting that the years of clinical work on B cell mediated cancers — which has had to grapple with the lethal cytokine storms that can engulf and extinguish patients — has significantly de-risked the safety aspects of their work.

It was Payne, the lead physician in the Autoimmune Blistering Clinic at Penn, who did the animal studies on their lead drug DSG3-CAART, providing the preclinical proof of concept they needed to set up the clinical studies. And they’ll have the full backing of Penn now that they’re shifting to human studies.

Steven Nichtberger

“During the past 18 months, Cabaletta has advanced a series of CAAR T cell therapy products, licensed foundational intellectual property and assembled leading scientists, clinicians and experts in the discovery, development, manufacturing and regulatory approval of cell therapy products to accelerate development of highly specific CAAR T cell therapies that may offer a potential one-time cure for certain B cell-mediated autoimmune diseases,” said Nichtberger.

Their A round came in last month, led by 5AM Ventures, with participation from the founders: Adage Capital Management, a “second large U.S.-based, healthcare-focused investment fund” and the University of Pennsylvania.


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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