Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations

NCTID	NCT06196827 (View at clinicaltrials.gov)
Description	The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. (Show More)
Development Status	Active
Indication	Inherited Retinal Dystrophy Associated With RPE65 Mutations
Disease Ontology Term	DOID:0110016
Compound Name	LX101
Compound Description	AAV2-RPE65
Sponsor	Innostellar Biotherapeutics Co.,Ltd
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	9
Results Posted	Not Available

Target Gene/Variant	RPE65
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
Editor Type	none
Dose 1	Undisclosed low dose (0.3mL/eye)
Dose 2	Undisclosed high dose (0.3mL/eye)
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	China