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ATSN 101

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Drug Profile

ATSN 101

Alternative Names: ATSN-101; SAR-439483

Latest Information Update: 14 Feb 2025

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At a glance

  • Originator University of Florida
  • Developer University of Florida; University of Pennsylvania
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference; Guanylate cyclase 1 replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Leber congenital amaurosis
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Phase I/II Leber congenital amaurosis

Most Recent Events

  • 06 Feb 2025 Atsena Therapeutics completes phase-I/II trial in Leber congenital amaurosis in USA (Subretinal) (NCT03920007)
  • 13 Nov 2024 ATSN 101 licensed for development and commercialization to Nippon Shinyaku in USA and Japan
  • 05 Sep 2024 Updated efficacy data and adverse event data from a phase I/II trial in Leber congenital amaurosis released by Atsena Therapeutics

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