Drugs / RS1 AAV Vector
last change Oct 2024 re-read 3 minutes ago

RS1 AAV Vector

also known as AAV-RS1 · AAV8-RS1 · AAV8-scRS/IRBPhRS

Gene therapy (AAV / viral vector) targets RS1

Developed for
retinoschisis · X-linked disease
Investigated by
National Eye Institute (NEI) · VegaVect, Inc.

Trials 1

2015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT02317887 Feb 2015 → Apr 2024 X-linked disease, retinoschisis VegaVect, Inc. Completed No outcome recorded

Evidence & citations 7 cited values

Every value below carries the sentence it was read from. 3 sources stand behind the page.

FieldValueCited text
Known as RS1 AAV Vector ClinicalTrials.gov intervention name — accepted as the source's own label NCT02317887 ↗
Known as AAV-RS1 “Objective: To evaluate the safety and tolerability of ocular AAV-RS1 vector (AAV8-scRS/IRBPhRS) gene transfer to the retina of participants affected with X-linked juvenile...” NCT02317887 ↗
Known as AAV8-RS1 “intravitreally administered AAV8-RS1 gene replacement therapy for XLRS participants” PMID 34390869 ↗ Aug 2021
Known as AAV8-scRS/IRBPhRS “Objective: To evaluate the safety and tolerability of ocular AAV-RS1 vector (AAV8-scRS/IRBPhRS) gene transfer to the retina of participants affected with X-linked juvenile...” NCT02317887 ↗
Modality Gene therapy (AAV / viral vector) “Adeno-associated virus vectors encoding RS1 gene” PMID 36227606 ↗ Oct 2022
Route Intravitreal “by intravitreal injection” NCT02317887 ↗
Target RS1 “introduce a healthy RS1 gene into eye cells” NCT02317887 ↗