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Laruparetigene zosaparvovec - Beacon Therapeutics

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Drug Profile

Laruparetigene zosaparvovec - Beacon Therapeutics

Alternative Names: AGTC-501; BIIB-088; Laru-zova - Beacon Therapeutics; rAAV2tYF-GRK1-RPGR

Latest Information Update: 22 Sep 2025

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At a glance

  • Originator Applied Genetic Technologies Corporation
  • Developer Beacon Therapeutics
  • Class Eye disorder therapies; Gene therapies
  • Mechanism of Action Gene transference; RPGR protein replacements
  • Orphan Drug Status

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

    Yes - Retinitis pigmentosa
  • New Molecular Entity No

Highest Development Phases

  • Phase II/III Retinitis pigmentosa

Most Recent Events

  • 19 Sep 2025 Phase-II clinical trials in Retinitis pigmentosa (In children, In adolescents, In adults) in USA (Subretinal) (NCT07174726)
  • 08 Jul 2025 Beacon Therapeutics plans regulatory submissions for Retinitis pigmentosa in USA and Europe
  • 08 Jul 2025 Beacon Therapeutics completes enrolment in phase-II/III VISTA trials in Retinitis pigmentosa (In adults, In adolescents, In children) in USA, United Kingdom, Australia (Intraocular) (NCT04850118)

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