Together We Thrive

Magenta Therapeutics is developing medicines intended to make cures possible for more patients. We do this by focusing on enabling more patients with severe or life-threatening diseases (blood cancers, genetic and autoimmune diseases) to benefit from the transformative potential of a curative stem cell transplant.

Stem cell transplant is a well-established and potentially curative medical procedure that can reset a patient’s blood and immune system for certain blood cancers, genetic diseases, or autoimmune diseases. Over 90,000 patients globally received a stem cell transplant in 2020 .

We know the patient journey involves more than just the patient – it involves family members, caregivers, health care providers, researchers and advocates, too. For questions or to request more information on resources available to patients and their families, please email us at

Clinical Trial Resources

Magenta is developing novel medicines to transform several key aspects of stem cell transplant for many diseases. Clinical trials allow us to evaluate investigational new treatments to generate the safety and efficacy data needed to obtain regulatory authorization to make these treatments available more broadly.  

  • A Phase 1/2 clinical trial, is currently ongoing to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential anti-leukemic activity and to establish the minimum safe and biologically-effective dose of a single dose of MGTA-117 in participants with relapsed/refractory (R/R) CD117+ acute myeloid leukemia (AML) and participants with myelodysplastic syndrome with excessive blasts (MDS-EB).
  • A Phase 2 clinical trial in collaboration with Bluebird Bio, Inc, is currently ongoing to characterize the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of MGTA-145 and plerixafor for hematopoietic stem cell (HSC) mobilization and apheresis in participants with sickle cell disease (SCD). Each party will characterize the collected cells and Magenta plans to gene-modify the cells and evaluate engraftment of cells in established translational models

For questions or to request more information, please email us at

Patient Organizations

Learn more about some of the organizations who are working on behalf of patients here:


For more information on the resources available to patients and their families, please email us at

Expanded Access Policy

Magenta also supports expanded access to
investigational products where there is substantial scientific evidence to
support the safety and the efficacy of a product for an indication when it is
logistically feasible and when permitted by applicable law.

Expanded access refers to the use of an
investigational therapy outside a clinical trial when the primary purpose is to
diagnose, prevent, or treat a serious condition in a patient. This is different
from a clinical trial, where more comprehensive safety and efficacy data are
collected. The intent of an expanded access program is to provide patients with
access to investigational medication for serious diseases or conditions where
there is no comparable or satisfactory alternative therapy available. We
understand the need for expanded access programs, and we recognize the
importance of having an appropriate policy.

Magenta will consider a number of factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies’ guidelines when reviewing requests for expanded access, including:

  • An independent request from a qualified and licensed treating physician submitted to that contains the requestor’s contact information as well as a description of the drug product requested, the disease to be treated, a rationale for the request, and any other relevant scientific and clinical information
  • The patient or patients to be treated have a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to treat the disease or condition including approved products or enrolling in a clinical trial
  • The potential patient benefit justifies the potential risks of the treatment use, and those risks are not unreasonable in the context of the disease or condition to be treated
  • Granting expanded access would not potentially compromise the scientific validity of broader development programs or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients

The above criteria are among those that Magenta will consider in determining whether to offer expanded access for individual use.  However, Magenta cannot make any guarantee that the investigational therapy will be available for a particular patient. All individual requests will be evaluated in a fair, unbiased manner, and we anticipate acknowledging receipt of requests sent to within five (5) business days.

At this time, Magenta believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapies. If you have additional questions, please speak with your physician or contact

Consistent with the 21st Century Cures Act, Magenta is publicly disclosing their policy on making investigational drugs available for expanded access and may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the expanded access record on after such record becomes active.