Press Release Details

Magenta Therapeutics to Present Preclinical Data on E478 Stem Cell Gene Therapy Expansion Program at the 2019 Annual Meeting of the American Society of Gene and Cell Therapy

April 29, 2019

-- Developing novel methods to expand gene-modified stem cells to achieve higher cell doses --

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 29, 2019-- Magenta Therapeutics (NASDAQ:MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, today announced the Company will highlight preclinical data on its E478 program in an oral presentation on Thursday, 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. Expansion may also increase the efficiency of manufacturing these therapies, which has proved to be a challenge with current approaches,” said Michael Cooke, Ph.D., Chief Scientific Officer, Magenta Therapeutics. “AHR antagonism is a clinically validated mechanism for expanding hematopoietic stem cells, as we have reported in Phase II studies with our MGTA-456 cell therapy that uses the same mechanism. Consistent with the E478 data presented to date, 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.”

Title: 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, Ph.D., Magenta Therapeutics
Presentation Date and Time:Thursday, May 2, 2019; 10:45 a.m. ET
Session Title: Engineered Cell Therapies
Room: Heights Courtyard 2

Forward-Looking Statement

This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions can be used to identify forward-looking statements.

The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities, including review under accelerated approval processes; orphan drug designation eligibility; regulatory approvals to conduct trials or to market products; whether Magenta's cash resources will be sufficient to fund Magenta's foreseeable and unforeseeable operating expenses and capital expenditure requirements; and other risks set forth under the caption “Risk Factors” in Magenta’s Registration Statement on Form S-1, as updated by Magenta’s most recent Annual Report on Form 10-K and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur.

Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Source: Magenta Therapeutics

Magenta Therapeutics:
Manisha Pai, Vice President, Communications & Investor Relations
617-510-9193
mpai@magentatx.com

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