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Cycle Pharmaceuticals

A UK-based biopharma company developing and commercializing treatments for rare genetic diseases.

Founded in 2012 and headquartered in Cambridge, with U.S. operations in Boston, the company emphasizes small-molecule therapeutics that address both metabolic and neurological conditions. Its portfolio targets a range of underserved indications, including urea cycle disorders, phenylketonuria, and hereditary spastic paraplegia.

In June 2025, Cycle received FDA approval for HARLIKU (nitisinone) tablets for alkaptonuria (AKU), a rare metabolic disorder characterized by buildup of homogentisic acid. HARLIKU is the first therapy specifically approved for AKU in the U.S., representing a milestone in the company’s expansion strategy. 

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