Magenta Therapeutics Announced It Will Present Clinical and Pre-Clinical Data from Across Immune and Blood System Reset Portfolio at European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting - Seite 2

Preclinical Data from Magenta’s Antibody-Drug Conjugate Conditioning Programs

Targeted, disease-modifying antibody-drug conjugates (ADCs) are designed to selectively and rapidly remove disease-causing cells in the body and enable immune and blood system reset and long-term engraftment, without the need for aggressive chemotherapy or radiation.

MGTA-117, the clinical candidate for ADC-based conditioning for stem cell transplant and gene therapy and Magenta’s most advanced conditioning program, is on track with IND-enabling toxicology studies ongoing and progress in GMP manufacturing. Magenta expects to generate initial clinical data in 2021.

Title: A Single Dose of Short Half-Life CD117 Antibody Drug Conjugate Enables Hematopoietic Stem Cell-Based Gene Therapy in Non-Human Primates (Abstract #O076)
Presenting Author: Rahul Palchaudhuri, Ph.D., Magenta Therapeutics, Cambridge, Mass.

This collaborative study with the National Institutes of Health (NIH) demonstrates that a single dose of a tool CD117-ADC molecule in non-human primates enables successful transplant and long-term engraftment of HSCs modified with a lentiviral vector encoding the β-globin gene, the gene that causes sickle cell disease and β-thalassemia. The ADC was well tolerated with none of the significant side effects that are seen with myeloablative dosing of busulfan chemotherapy.

Title: A Novel Targeted Approach to Achieve Immune System Reset: CD45-Targeted Antibody-Drug Conjugates Ameliorate Disease in Preclinical Autoimmune Disease Models and Enable Auto HSCT (Abstract #O030)
Presenting Author: Geoff Gillard, Ph.D., Magenta Therapeutics, Cambridge, Mass.

Magenta’s CD45-ADC program targets CD45, a protein expressed on immune cells and blood stem cells, and is designed to remove the cells that cause autoimmune diseases to enable curative immune reset. Magenta has identified a lead antibody for this program and IND-enabling work on CD45-ADC is progressing in 2020.

Preclinical data in this abstract show that a single dose of CD45-ADC removed disease-causing T-cells, enabling successful immune reset to halt disease progression and was well tolerated in three models of autoimmune disease: multiple sclerosis, systemic sclerosis and inflammatory arthritis.

Title: A CD45-Targeted Antibody Drug Conjugate Enables Allogeneic Hematopoietic Stem Cell Transplantation as a Single Agent in Mice (Abstract #A174)
Presenting Author: Sharon Hyzy, M.S., Magenta Therapeutics, Cambridge, Mass.