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2025 Oxford-Harrington Rare Disease Scholar Award
Hematology, Immunology, Musculoskeletal, Reproductive
Autoantigen-Specific T Cell Therapies for the Prevention and Treatment of Antiphospholipid Syndrome
2023 Harrington Scholar-Innovator
Antiphospholipid syndrome (APS) is a systemic autoimmune disorder that causes blood clots and pregnancy complications, including repeated miscarriages. Existing treatments—commonly lifelong therapy with blood thinners—do not address the underlying autoimmune problem and often fail. Rheumatologist, Maximilian Konig, MD, and his team have developed a way to reprogram the immune system to stop the autoimmune attack without suppressing its vital functioning.
The groundbreaking approach, called CATCR (chimeric autoantigen T-cell receptor, pronounced “catcher”), involves immune cell engineering similar to CAR-T cell technology— except that the scientists genetically edit the T-cell receptor directly, turning them into precise weapons against rogue B cells that drive APS.
“In patients suffering from APS, a small subset of B cells becomes self-reactive—they recognize and attack an abundant plasma protein called beta-2-glycoprotein I (B2GPI),” explains Dr. Konig. “These rogue B cells also make the autoantibodies that cause blood clots and pregnancy loss in APS. Armored with CATCRs, we have shown that a very small number of re-engineered T-cells can eliminate the B2GPI-reactive B cells while sparing all normal B cells to fight infection.”
With Harrington support, Dr. Konig is refining structural characteristics and functions of CATCR-T cells while conducting efficacy and safety studies in humanized models.
“Having a team of experts in drug manufacturing, regulatory affairs, and commercial development is a major advantage in advancing CATCR,” Dr. Konig says. “We hope to leverage this technology to treat patients with APS first, and eventually design precision cellular therapies for various other autoimmune and rheumatic diseases.”