According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of inherited metabolic disorders (IMDs) at a conference on IMD held in Orlando, Florida.
About the Trial
The MGTA-456 stem cell therapy provides hematopoietic stem cells (HSC) that are carefully matched to each patient. HSCs are immature cells found in blood and bone marrow that develop into many different types of blood.
About Inherited Metabolic Disorders
IMDs are inherited conditions resulting in metabolism issues. The majority of patients with IMDs present a defective gene resulting in enzyme deficiency. There are many IMDs and treatments, symptoms, and prognoses. Here are three examples.
- Adrenoleukodystrophy: a hereditary condition that damages the membrane that covers the brain’s nerve cells.
- Hurler syndrome or mucopolysaccharidosis type 1(MPS1): caused by mutated genes on chromosome 4 with a resulting deficiency in lysosomal enzymes. Usually occurring in infants 3-6 months.
- Metachromatic leukodystrophy: causes the accumulation of fats called sulfatides in cells that damage brain tissue, causing deterioration of intellectual and physical functions.
IMDs are rare, progress rapidly, and may be fatal if not treated early.
A Phase II trial involving patients who have blood cancers diagnosed as high risk is being conducted by a team at the University of Minnesota’s Masonic Cancer Center. The team is studying the cryopreserved (cooled to very low temperatures) formulation of MGTA-456.
Magenta’s Chief Medical Officer said that after one year the results of the trial showed rapid improvement and a halt in disease progression. He said the data are especially encouraging as the same results have not been seen in treatments that are currently available to patients or with other gene therapies now being studied.
Two patients participated in the trial. The company is in the process of enrolling additional patients by year’s end.
In 2019, the FDA granted MGTA-456 the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of IMDs. Magenta intends to continue to work with the FDA as part of the RMAT designation. The focus will be on a registration-enabling study.
Discussions with the EMA will be under way shortly regarding European development of MGTA-456.
About the Company
Magenta Therapeutics was co-founded in 2015 with the intent to revolutionize stem cell transplant for blood-based and autoimmune diseases. Magenta’s goal is to make one-time therapies available to more patients and provide a higher quality of replacement stem cells.
Magenta is committed to finding areas with unmet needs. The company is a pioneer in its determination to make the process safer, more effective, and easier.
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