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Waltham biopharmaceuticals firm Repligen Corp. has filed an investigational new drug (IND) application with the U.S. Food and Drug Administration for a Phase 1 study of its drug candidate to treat Friedreich’s ataxia, a rare neurodegenerative disease.
The IND is for Repligen’s RG2833, a selective histone deacetylase 3 inhibitor, and the Phase 1 study is intended to look at the safety and effects of the drug candidate.
Friedreich’s ataxia is a congenital neurodegenerative disease caused by a single gene defect that leads to the body not producing enough of the protein frataxin, leading to progressive damage to the nervous system and loss of muscle function.
The filing follows an announcement in December that Repligen (Nasdaq: RGEN) had won a $731,000 Muscular Dystrophy Association research grant for the development of its Friedreich’s ataxia treatment.
According to a news release, Repligen has been financed with grants from the Muscular Dystrophy Association, the Friedreich’s Ataxia Research Alliance, GoFAR and the National Ataxia Foundation.
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