Drugs / AGTC-501
last change Jun 2026 re-read 3 minutes ago

AGTC-501

Gene therapy (AAV / viral vector) targets RPGR

Developed for
retinitis pigmentosa
Investigated by
Beacon Therapeutics

Trials 3

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 23 trials
Phase 2 NCT07174726 Sep 2025 → Dec 2029 expected retinitis pigmentosa Beacon Therapeutics Active not recruiting No outcome recorded
Phase 2 NCT06275620 Nov 2023 → Nov 2025 overdue retinitis pigmentosa Beacon Therapeutics Enrolling by invitation No outcome recorded
Phase 2 NCT06333249 Apr 2021 → Apr 2023 overdue retinitis pigmentosa Beacon Therapeutics Active not recruiting No outcome recorded
Comparator or background therapy1 trial
Phase 2/3 NCT04850118 Comparator Mar 2024 → Jun 2026 overdue retinitis pigmentosa Beacon Therapeutics Active not recruiting No outcome recorded

Evidence & citations 7 cited values

Every value below carries the sentence it was read from. 4 sources stand behind the page.

FieldValueCited text
Known as AGTC-501 “The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to...” NCT07174726 ↗
2

“Each participant will receive the assigned dose of AGTC-501 in one eye on a single occasion.” NCT06333249 ↗

NCT06275620 ↗

Known as laruparetigene zovaparvovec “This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501/laruparetigene zovaparvovec ))...” NCT04850118 ↗
1

“The purpose of this Phase 2 Study is to see if the investigational study drug, laruparetigene zovaparvovec, also known as AGTC-501, given in both eyes, is safe and works to...” NCT07174726 ↗

Known as rAAV2tYF-GRK1-hRPGRco ClinicalTrials.gov intervention name — accepted as the source's own label NCT04850118 ↗
Known as rAAV2tYF-GRK1-RPGR “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...” NCT06333249 ↗
1

“AGTC-501 (rAAV2tYF-GRK1-RPGR)” NCT06275620 ↗

Modality Gene therapy (AAV / viral vector) “recombinant adeno-associated virus vector” NCT04850118 ↗
Route Other “treated by subretinal injection” NCT07174726 ↗
Target RPGR “Adeno-associated virus vector expressing a human RPGR gene” NCT04850118 ↗