Company intelligence / Profile preview

Cyprio

Miami, United States

Ownership: Private
Development stage: Other

Overview

CUTX-101 (copper histidinate) NDA received a Complete Response Letter in October 2025 due to cGMP issues at the manufacturing facility; no concerns on efficacy or safety data. Positive topline results showed improved survival with early treatment. AAV-ATP7A gene therapy in pre-clinical development.[2][3]

Therapeutic areas: Rare DiseasesMetabolic Disorders
Modalities: Small moleculeGene therapy
Industry: Biotech

Partnerships

Sentynl TherapeuticsNICHD (NIH)

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