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 Participants With X-linked Retinitis Pigmentosa
Latest Information Update: 03 Feb 2025
At a glance
- Drugs Laruparetigene zovaparvovec (Primary)
- Indications Retinitis pigmentosa
- Focus Adverse reactions
- Acronyms DAWN
- Sponsors Beacon Therapeutics
- 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.
- 06 Dec 2024 Interim results presented in a Beacon Therapeutics media release
- 06 Dec 2024 According to Beacon Therapeutics media release, today announced presentation of 3-month interim safety and efficacy results of the Phase 2 DAWN trial in patients with XLRP at the FLORetina-ICOOR Meeting 2024 in Florence, Italy.