Significant needs remain for patients living with autoimmune diseases, blood cancers, and rare genetic diseases. Bone marrow transplant is a potential cure for many, but its high risks, toxic side effects and complexity prevent many patients from being able to benefit. Magenta is working comprehensively across the transplant journey to change this reality.
Transplant Patient Journey
We aim to address significant needs across all aspects of transplant medicine. By pioneering an innovative portfolio of novel therapeutics designed to make the entire bone marrow transplant process more effective, safer and easier, we aim to unleash the full potential of bone marrow transplant for those living with a wide array of conditions such as multiple sclerosis, scleroderma, acute myeloid leukemia, myelodysplastic syndromes, inherited metabolic diseases, and sickle cell disease.
The Need in Autoimmune Disease
People with severe autoimmune disorders have a limited set of current therapies to manage their disease, none with the curative potential of bone marrow transplant. Patients face significant shortcomings in treatment, including:
- In some autoimmune disorders such as scleroderma, no disease-modifying therapies currently exist
- Even where therapies exist, patients still progress in disease severity
- Despite the curative potential of bone marrow transplant, the mortality and morbidity risk is currently unacceptable to many patients and physicians
We believe our therapeutics will transform transplant for autoimmune diseases in the following areas:
There has been tremendous success in the use of bone marrow transplants for blood cancers. However, challenges remain:
- Many patients who could benefit from transplant are ineligible due to older age or poor health
- Half of all patients with acute myeloid leukemia (AML) relapse after transplant
- Transplant-related mortality remains high, and morbidities are significant and long term
- The risk of acute and chronic graft-versus-host disease (GVHD) remains high
We believe our therapeutics will transform transplant for blood cancers in the following areas:
Inherited Metabolic Disorders, Bone Marrow Failure, Hemoglobinopathies
Wider use of transplant may help transform the treatment of rare diseases, such as inherited metabolic disorders, bone marrow failure, and hemoglobinopathies such as sickle cell disease. These diseases represent areas of significant unmet patient need, where improvements in transplant medicine could usher in a new era of treatment.
Current disease management often involves intense chronic treatments, and in some cases, there are currently no treatment options.
- Preparations for transplant can be associated with many long-term effects, such as new cancers and infertility
- There is difficulty finding suitable matches in patients with life-threatening conditions, resulting in high rates of graft rejection and post-transplant complications like GvHD
- Patients can experience delayed engraftment of stem cells and immune recovery
- Chronic GvHD is a real risk, resulting in some patients trading one chronic disease for another
Our portfolio has the potential to transform the following aspects of transplant for rare diseases: