TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.

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Grant Award Details

Grant Number:
CLIN1-14840
Investigator(s):
Institution:
Type:
PI

Award Value:
$4,000,000
Status:
Pre-Active

Grant Application Details

Application Title:

TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.

Public Abstract:
Therapeutic Candidate or Device

An allogenic, off the shelf, engineered regulatory T cell product that mimics the function of T regulatory Type 1 (Tr1) cells.

Indication

Prevention of acute and chronic Graft versus Host Disease (GvHD) in patients undergoing mismatched stem cell transplant.

Therapeutic Mechanism

GvHD is driven by the reaction of allogenic donor T cells to host target tissues and antigens. TRX103 will suppress GvHD by secretion of IL-10 and other cytokines in target tissues and organs, as well as through the stimulation of production of de-novo Tr1 cells in patients.

Unmet Medical Need

GvHD affects patients undergoing mismatched hematopoietic stem cell transplant (HSCT) and is a major cause of morbidity and mortality. Only ~50% of HSCT patients are disease and relapse-free (GRFS) after one year. TRX103 will broaden access to transplants, while reducing the burden of GvHD.

Project Objective

IND submission completed.

Major Proposed Activities

  • Preclinical in vitro and in vivo studies to characterize the TRX103 product and determine its safety and efficacy in preclinical models of disease.
  • Process development, optimization and technology transfer to partner CDMO for production of the TRX103 product to enable IND submission.
  • Process Development, engineering and clinical manufacturing runs of the TRX103 product to enable IND submission.
Statement of Benefit to California:
Mismatched hematopoietic stem cell transplants have worse outcomes than matched transplants, due to higher incidence and severity of GvHD. The proposed research will enable human trials of a novel product, TRX103 in these patients. Should the research achieve its objective, Californians will have access to a therapy that will broaden access to curative stem cell transplants, while reducing the (significant) risks associated with GvHD.