Patients & Families
Our Shared Mission
The science of bone marrow transplant is opening new horizons in the treatment of a wide range of diseases — including many where transplant would have been unthinkable.
Meet one of the people who inspire the Magenta team in their quest to change medicine.
The bone marrow transplant journey involves family members, caregivers, researchers, as well as advocates. Learn more about some of the organizations who are working on behalf of patients here:
Be the Match, a non-for profit organization operated by the National Marrow Donor Program NMDP, serves as a critical link between stem cell donors and recipients. Learn more about their vital work, here: bethematch.org
- American Society of Hematology
- Center for International Blood and
Marrow Transplant Research®
- Blood & Marrow Transplant
- American Society for Blood and
- The Bone Marrow Foundation
- European Society for Blood and
- Leukemia and Lymphoma Society
For more information on the resources available to patients and their families, please email us at firstname.lastname@example.org.
Clinical Trial Resources
Magenta Therapeutics is addressing all aspects of bone marrow transplant through innovative and targeted drug approaches. For more information about our clinical trials, email us at email@example.com.
To learn more about our ongoing Phase 2 study of MGTA-456 cell therapy in inherited metabolic disorders, please visit: https://www.clinicaltrials.gov/ct2/show/NCT03406962?term=MGTA-456&rank=1.
To learn more about our ongoing Phase 1 study of MGTA-145 (M100) stem cell mobilization program in healthy volunteers, please visit: https://www.clinicaltrials.gov/ct2/show/NCT03932864?term=MGTA-145&rank=1.
Expanded Access Policy
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. Information about Magenta’s currently enrolling clinical trials can be found at clinicatrials.gov.
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 firstname.lastname@example.org that contains the requestor’s contact information as well as a description of the drug product requested, the disease to be a 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 email@example.com 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 firstname.lastname@example.org.
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 clinicaltrials.gov after such record becomes active.