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A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene

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Trial Profile

A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 03 Feb 2025

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At a glance

  • Drugs Laruparetigene zovaparvovec (Primary)
  • Indications Retinitis pigmentosa
  • Focus Therapeutic Use
  • Acronyms Skyline
  • Sponsors Beacon Pharmaceuticals

Most Recent Events

  • 28 Jan 2025 According to Beacon Therapeutics media release, company today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for the treatment of X-linked retinitis pigmentosa (XLRP). The FDA RMAT designation for laru-zova recognizes preliminary clinical evidence from the Phase 2 DAWN and SKYLINE trials.
  • 15 Oct 2024 According to Beacon Therapeutics media release, the company announced presentation of 24-month interim safety and efficacy results of the Phase 2 SKYLINE trial in patients with XLRP at the American Academy of Ophthalmology's Annual Meeting in Chicago, Illinois.
  • 15 Oct 2024 Interim results presented in the Beacon Therapeutics Media Release.

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