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Magenta To Present Preclinical Data From Stem Cell Gene Therapy Expansion Program.

CAMBRIDGE, Mass., April 29, 2019 -- Magenta Therapeutics (MGTA) announced it will present preclinical data on its small molecule/stem cell expansion program in an oral presentation on May 2 at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT) in Washington, D.C.

Magenta is developing E478 to achieve high doses of gene-modified stem cells for better outcomes in patients with genetic disorders, including sickle cell disease and thalassemia, where viral vector or gene editing technologies are used to correct stem cells.

E478 is a novel and proprietary small molecule that uses AHR antagonism to expand gene-modified hematopoietic stem cells during manufacture, then the corrected cells are infused as the medicine for the patient.

Magenta intends to develop E478 in partnership with gene therapy companies.

Stem cell gene therapy and genome editing are promising approaches for treating genetic disorders.

However, these therapies are most effective when a large dose of gene-modified cells is administered by a stem cell transplant, as cell dose is crucial for patient outcomes, the company said.

Expansion may also increase the efficiency of manufacturing these therapies, which has proved to be a challenge with current approaches.

AHR antagonism is a clinically validated mechanism for expanding hematopoietic stem cells, as we have reported in Phase 2 studies with the company's MGTA-456 cell therapy that uses the same mechanism.

"Consistent with the E478 data presented to date," said CSO Michael Cooke, "we believe the results for ASGCT show that AHR antagonism is a promising method for expanding gene-modified stem cells while maintaining the editing or transduction rates, which will be important for patients receiving these therapies."

Presentation: A Novel Aryl Hydrocarbon Receptor Antagonist Expands Adult Human Hematopoietic Stem Cells from Mobilized Peripheral Blood and Bone Marrow and Increases the Dose of CRISPR/Cas9 Gene-Edited NSG-Repopulating Cells, Abstract #979

Presenter: Megan Hoban, Magenta Therapeutics

Presentation Date: May 2, 2019

Session Title: Engineered Cell Therapies


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Title Annotation:Preclinical Research
Publication:Stem Cell Lab World
Date:Apr 29, 2019
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