CAR T Cell Therapies Show Promise in Treating Autoimmune Diseases

The pharmaceutical industry is witnessing a significant shift as CAR T cell therapies, previously successful in cancer treatment, are now being leveraged to combat autoimmune disorders. Several companies are at the forefront of this innovative approach, with early to mid-stage clinical trial readouts expected throughout 2025.

Breakthrough Potential in Lupus and Beyond

Bristol Myers Squibb (BMS) is leading the charge with its CD19 NEX-T therapy, building on its expertise in cancer-focused CAR T treatments. The company's Phase I Breakfree-1 and Breakfree-2 studies in systemic lupus erythematosus (SLE) have shown promising early results. Three patients followed for at least six months were able to discontinue all other therapies without new signs of disease activity, with one patient achieving disease remission at the six-month mark.

Rosanna Ricafort, Vice President and Head of Late Development, Hematology and Cell Therapy at BMS, stated, "Our ultimate goal is to target the root cause of disease and drive long-term, treatment-free disease control for patients with severe forms of these autoimmune diseases."

Innovative Approaches to Improve Tolerability and Accessibility

Kyverna Therapeutics is making strides with KYV-101, a CD19-targeted CAR T therapy designed to avoid cytokine release syndrome and enhance tolerability. The company is targeting three conditions: stiff person syndrome (SPS), myasthenia gravis, and lupus nephritis. Kyverna expects to complete enrollment for its Phase II SPS trial by mid-2025, with topline results anticipated in early 2026.

CRISPR Therapeutics is focusing on the development of CTX112, an off-the-shelf allogeneic CAR T therapy. This approach aims to overcome the limitations of autologous treatments, such as high costs and limited availability. The company has expanded its basket trial to include systemic sclerosis and inflammatory myositis, with updates expected mid-2025.

Emerging Players and Novel Mechanisms

Autolus Therapeutics' obecabtagene autoleucel (obe-cel), already FDA-approved for certain leukemia patients, is being evaluated for SLE. The therapy's unique "fast-off" kinetic mechanism is designed to provide more controlled T cell activation. Initial data from the Phase I CARLYSLE study in advanced SLE are expected in the first quarter of 2025.

Cabaletta Bio's resecabtagene autoleucel (rese-cel) incorporates a 4-1BB domain to enhance T cell activity. The company's RESET program is assessing rese-cel across multiple autoimmune disorders, with promising early results in myositis, SLE, and systemic sclerosis. Cabaletta anticipates several milestones in 2025, including additional clinical readouts and discussions with the FDA regarding a potential registrational path.

As these therapies progress through clinical development, the pharmaceutical industry is poised for potentially transformative treatments that could offer long-term, drug-free remission for patients with autoimmune diseases. The coming year will be crucial in determining the future of CAR T cell therapies beyond oncology and their potential to revolutionize autoimmune disease management.