Pharma Focus Europe

TR1X Receives FDA Approval for TRX103: Allogeneic Regulatory T-Cell Therapy Targeting Autoimmune Diseases

Thursday, April 11, 2024

Tr1X, Inc., a company focusing on autoimmune and inflammatory disease treatments, has received FDA approval for its Investigational New Drug (IND) application for TRX103. This drug aims to prevent Graft versus Host Disease (GvHD) in patients undergoing HLA-mismatched hematopoietic stem cell transplantation (HSCT). The company plans to begin a Phase 1 study of TRX103 in the second quarter of 2024, focusing on its potential as an allogeneic off-the-shelf Tr1 Treg therapy.

Maria Grazia Roncarolo, M.D., Co-Founder, President, and Head of R&D at Tr1X, noted the significance of this milestone, highlighting TRX103 as the first engineered allogeneic Tr1 regulatory T cell product. She emphasized its potential in providing proof-of-concept data swiftly while the company continues its development for various autoimmune and inflammatory diseases, including Crohn's disease.

TRX103 differs from autologous Tr1 cells due to its feasibility and cost-effectiveness. It's an off-the-shelf product with distinct biological properties compared to other Treg and CAR-T cell therapies, showing promise in reducing inflammation, suppressing pathogenic cells, and resetting the immune system. The scalable production process yields billions of cells efficiently, facilitating the development of pipeline candidates for broader patient populations with unmet medical needs.

Dr. Monzr M. Al Malki, lead investigator of the Phase 1 study, highlighted the urgent need for innovative treatments in allogeneic stem cell transplantation to address the burden of morbidity and mortality related to GvHD and its complications.

TRX103, derived from healthy donor CD4+ cells and engineered to mimic Tr1 regulatory T cell function, is being developed for various immune and inflammatory disorders. Preclinical models have demonstrated its tolerability, efficacy, and potential to reset immune systems. TRX103 aims to overcome limitations of current autoimmune disease cell therapies, including limited persistence and adverse effects like cytokine release syndrome (CRS) and neurotoxicity.



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