Together We Thrive

Magenta Therapeutics is developing medicines to make cures possible for more patients, advancing a unique and fully integrated platform of therapies designed to reset the immune system through stem cell transplant enabling more patients to live free of their diseases. 

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 all 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 approval of these treatments. 

Current Clinical Trials:

Learn more about our ongoing Phase 2 study of MGTA-456 cell therapy in inherited metabolic disorders.

• MGTA-456 is a cell therapy available in a clinical trial and designed to rapidly halt disease progression of inherited metabolic disorders.

• Currently enrolling patients in a Phase 2 clinical trial, IMD-001. The primary goal of the IMD-001 study is to assess the number of patients in whom MGTA-456 successfully engraft, as well as the speed of engraftment.

• Engraftment is when the cells received from the transplant move through the bloodstream into the bone marrow and begin to grow and make new blood and immune cells. Patients between the ages of 6 months to 16 years with Hurler syndrome (MPS-1), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or Krabbe disease (globoid cell leukodystrophy, GLD) are eligible.

• Additional details and information on how to connect with a study center can be found here.

Learn more about our ongoing Phase 1 study of MGTA-145 stem cell mobilization program in healthy volunteers.

• 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.