Francisco Conesa Buendía: CAR-T and Treg Cell Therapies Transforming Autoimmune Disease Treatment
Francisco Conesa Buendía, Assistant, Cell and Gene Therapies Manufacturingat Memorial Sloan Kettering Cancer Center, shared on LinkedIn:
“From Promise to Practice: CAR-T and Treg Cell Therapies Transforming Autoimmune Disease Treatment
Autoimmune diseases, impacting millions globally, are seeing a paradigm shift with the advent of Chimeric Antigen Receptor (CAR-T) and Regulatory T cell (Treg) therapies. These cutting-edge approaches promise targeted, long-term remission while addressing the limitations of traditional treatments.
Key Innovations Driving the Future
1. CAR-T Therapy for Autoimmunity:
CD19-Targeted CAR-T Cells:
– Delivered 100% remission in refractory SLE for up to 29 months.
– Ongoing trials led by Friedrich Alexander University, Kyverna Therapeutics, Adicet Bio, and Cabaletta Bio are expanding indications to include lupus nephritis, systemic sclerosis, and idiopathic inflammatory myopathies.
BCMA-Targeted CAR-T Cells:
– Achieved 92% drug-free remission in patients with neuromyelitis optica spectrum disorder (NMOSD) in studies conducted by Cartesian Therapeutics and IASO Bio.
– Trials by Sana Biotechnology and Immpact Bio explore combined CD19 and BCMA targeting for enhanced B-cell depletion.
– Bi-Specific CAR-Ts:
– Innovative designs by Gracell Bio, Novartis, and iCell Gene Therapeutics combine CD19 and BCMA targeting, reducing the risk of antigen escape.
2. Regulatory T Cell (Treg) Therapies:
FOXP3+ Treg Cells:
– Trials led by Quell Therapeutics and Sangamo Therapeutics are testing HLA-A2-specific CAR FOXP3+ Tregs for use in liver and kidney transplantation.
– Sonoma Biotherapeutics is advancing CAR FOXP3+ Tregs for rheumatoid arthritis and hidradenitis suppurativa.
Tr1 Therapies:
– Engineered Tr1 therapies by Tr1X Bio and ActiTrexx demonstrate immune tolerance and suppression of inflammation in Crohn’s disease and GvHD.
Clinical Results and Trials
Recent Successes:
– SLE: 100% remission in patients treated with CD19-targeted CAR-T cells led by Kyverna Therapeutics.
– NMOSD: 92% relapse-free outcomes in patients treated with BCMA-targeted CAR-T by #IASOBio.
– Myasthenia Gravis: Significant improvement in mobility in trials conducted by Cartesian Therapeutics.
Active Trials:
– SLE, Lupus Nephritis: Bi-specific CAR-T therapies (NCT05474885, NCT05846347).
– Myasthenia Gravis, Systemic Sclerosis: Anti-BCMA CAR-T (NCT04146051, NCT06328777).
– Rheumatoid Arthritis and Hidradenitis Suppurativa: Treg therapies by Sonoma Biotherapeutics (NCT06201416, NCT06361836).
Collaborating Companies
Companies leading this charge include:
– Kyverna Therapeutics
– Cartesian Therapeutics
– Quell Therapeutics
– Sonoma Biotherapeutics
– Sangamo Therapeutics, Inc.
– Adicet Bio, Inc
– Cabaletta Bio
– Gracell Biotechnologies
– Sana Biotechnology, Inc.
– Tr1X
– ActiTrexx GmbH
Why This Matters
These therapies herald a new era of precision medicine by offering targeted, lasting solutions for complex autoimmune diseases.
For more information, see paper below.”
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