Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients

NCTID	NCT06066008 (View at clinicaltrials.gov)
Description	This trial is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye. (Show More)
Development Status	Active
Indication	X-linked Retinoschisis
Disease Ontology Term	DOID:0060763
Compound Name	ZM-01
Compound Description	rAAV-hRS1
Sponsor	Zhongmou Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	9
Results Posted	Not Available

Target Gene/Variant	RS1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type
Dose 1	2.07E11 vg/eye
Dose 2	Undisclosed high dose
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China