Tr1X, Inc. (Trix), a clinical-stage biopharmaceutical company, announced that the California Institute for Regenerative Medicine (CIRM) has awarded the company an $8 million grant to support its ongoing phase 1/2a clinical trial of TRX103, an engineered Tr1 Treg cell therapy for use in the prevention of graft-versus-host disease (GvHD) in blood cancer patients undergoing mismatched stem cell transplants. This award follows a $4 million CIRM grant received by Tr1X in early 2024 for late-stage preclinical research that helped to advance TRX103 to human trials.
The TRX103 phase 1/2a clinical trial is being led by Maria Grazia Roncarolo, MD, co-founder, president and head of R&D at Tr1X, with patients enrolling at leading stem cell transplant centres across the country. The trial is designed to treat patients receiving a mismatched or haploidentical transplant with a one-time infusion of TRX103. The company has reported positive initial persistence and safety data in the first two patient cohorts, with additional data anticipated to be reported in 2025. TRX103, which is the company’s lead program, is also being tested in a phase 1/2a trial for patients with treatment-refractory Crohn’s disease.
“With CIRM’s support, TRX103 has rapidly advanced from promising preclinical findings into clinical trials, bringing us closer to transforming mismatched stem cell transplants into safer, life-saving options,” said Dr. Roncarolo. “By harnessing the unique power of engineered Tr1 cells, our goal is to prevent graft-versus-host disease, improve immune reconstitution and induce tolerance to host cells—therefore expanding access to curative therapies for patients who urgently need them.”
GvHD occurs when donor cells attack the recipient’s tissue and organs, often causing life-threatening complications. Current treatments for GvHD, which suppress the immune system, can lead to infections and hinder cancer treatment effectiveness. TRX103, an allogeneic engineered regulatory T cell product, may improve transplant access for patients without a matched donor while reducing GvHD risks.
Dr. Roncarolo added, “This trial represents a hope for all of us transplanters—not only for better outcomes but also for a future where effective, personalized treatments like hematopoietic stem cell transplant become accessible to all patients, regardless of donor match.”
TRX103 is an investigational allogeneic off-the-shelf engineered T cell product generated from CD4+ cells sourced from healthy donors. These CD4+ cells are engineered to become cells that mimic the function of type 1 regulatory (Tr1) cells, called TRX cells. Tr1X is developing TRX103 for the treatment of several immune and inflammatory disorders. Multiple preclinical studies have shown TRX103 to be tolerable and effective and to have the potential to reset the immune system to a healthy state. TRX103 has the potential to overcome major limitations of current cell therapies for autoimmune diseases, which include limited persistence and side effects including cytokine release syndrome (CRS) and neurotoxicity.
Tr1X is a clinical-stage private biotechnology company pioneering a new class of curative cell therapies designed to fundamentally reset the immune system in patients with autoimmune and inflammatory diseases.
