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Magenta Therapeutics cell therapy gets US FDA regenerative medicine tag

Magenta Therapeutics Inc. said the U.S. Food and Drug Administration granted a regenerative medicine advanced therapy designation to its experimental one-time cell therapy MGTA-456 to treat multiple inherited metabolic disorders.

The designation allows for expediting the drug development and review processes for therapies that show the potential to treat serious or life-threatening diseases or conditions.

The biotechnology company is developing MGTA-456 for a number of inherited disorders and is evaluating the drug in a phase 2 study in patients older than six months with Hurler syndrome, cerebral adrenoleukodystrophy, metachromatic leukodystrophy or globoid cell leukodystrophy.

Hurler syndrome is a progressive genetic disease caused by the deficiency of a certain enzyme, alpha-L-iduronidase. Symptoms of the disease include developmental delay, cognitive decline and joint stiffness. It also affects the heart and respiratory system. During the trial, MGTA-456 showed recovery of deficient enzyme levels in the blood of patients with Hurler syndrome as early as 42 days after the treatment.

Cerebral adrenoleukodystrophy affects boys between four and 10 years old and leads to permanent disability and death usually within four to eight years. Magnetic resonance imaging of the patients in the trial showed resolution of inflammation in the brain 28 days following treatment.

Patients with metachromatic leukodystrophy experience deterioration of intellectual functions and motor skills, such as the ability to walk. Other symptoms include seizures, paralysis, an inability to speak, blindness and hearing loss. These patients eventually lose awareness and become unresponsive.

MGTA-456 provides a high dose of stem cells that assist in the regeneration of the patient's immune system.

Cambridge, Mass.-based Magenta develops novel medicines to improve stem cell transplants.

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