Repligen Corporation has filed an Investigational New Drug Application with the US Food and Drug Administration (FDA) for a phase 1 study of what could be the first drug to treat the deadly genetic disease Friedreich’s ataxia.
The trial will consist of a double-blind, single ascending dose phase 1 study to evaluate the pharmacokinetic and safety profile of RG2833 in up to 40 healthy subjects.
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Repligen Corporation president and CEO Walter C Herlihy said that RG2833 is an orally bioavailable compound that targets activation of the defective gene responsible for Friedreich’s ataxia.
“If this therapeutic approach is successful, it has the potential to change progression of the disease and significantly impact patients’ lives,” Herlihy said.
Friedreich’s ataxia is an inherited neurodegenerative disease caused by a single gene defect resulting in inadequate production of the protein frataxin, which leads to degeneration of the nerves controlling muscle movements in the arms and legs and the nerve tissue in the spinal cord.
Preclinical studies in animal models and patient cells have shown that RG2833 crosses the blood brain barrier, activates the defective frataxin gene and increases production of the protein frataxin.
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