Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene

NCTID	NCT03328130 (View at clinicaltrials.gov)
Description	The study is a Phase I/II, monocentric, open-label, dose-ranging safety and efficacy gene therapy intervention by subretinal administration of AAV2/5-hPDE6B. At least twelve patients 18 years of age or older, within four consecutive cohorts of patients, will be recruited. Then at least four patients 13 years of age or older, within a fifth cohort, will be recruited. (Show More)
Development Status	Active
Indication	Retinitis Pigmentosa
Disease Ontology Term	DOID:0110375
Compound Name	CTX-PDE6B
Compound Description	HORA-PDE6B
Sponsor	eyeDNA Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	23
Results Posted	Not Available

Target Gene/Variant	PDE6B
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2/5
Editor Type	none
Dose 1	3.4E11 vg/eye
Dose 2	6.4E11 vg/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	France