rAAV2.REP1

also known as AAV2-REP1

Developed for: choroideremia
Investigated by: University of Oxford · Alberta Innovates Health Solutions · Canada Foundation for Innovation · Canadian Institutes of Health Research (CIHR) · Choroideremia Research Foundation Canada · Foundation Fighting Blindness
+11 more
· Imperial College London · Manchester University NHS Foundation Trust · Moorfields Eye Hospital NHS Foundation Trust · Oxford University Hospitals NHS Trust · STZ eyetrial · University College, London · University Hospital Southampton NHS Foundation Trust · University Hospital Tuebingen · University of Alberta · University of Manchester · University of Southampton

2012201320142015201620172018201920202021202220232024202520262027

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 1/22 trials
Phase 1/2	NCT02077361	Apr 2015 → Aug 2017	choroideremia	University of Alberta	Completed	No outcome recorded
Phase 1/2	NCT01461213	Oct 2011 → Oct 2017	choroideremia	University of Oxford	Completed	No outcome recorded
Phase 21 trial · 1 missed
Phase 2	NCT02671539	Jan 2016 → Feb 2018	choroideremia	STZ eyetrial	Completed	Missed primary

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

Field	Value	Cited text
Known as	rAAV2.REP1	ClinicalTrials.gov intervention name — accepted as the source's own label NCT02077361 ↗ 2 NCT01461213 ↗ NCT02671539 ↗
Known as	AAV2-REP1	“OBJECTIVE: To assess the safety and efficacy of retinal gene therapy with an adeno-associated virus vector (AAV2) designed to deliver a functional version of the CHM gene...” PMID 31465092 ↗ Nov 2019
Action	Restore	“replaces the defective gene with a normal copy” NCT02077361 ↗
Modality	Gene therapy (AAV / viral vector)	“Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein-1 (REP1)” NCT02077361 ↗
Route	Other	“Patients will receive a subretinal injection” NCT02077361 ↗
Target	CHM	“functional version of the CHM gene” PMID 31465092 ↗ Nov 2019