Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients

NCTID	NCT06292650 (View at clinicaltrials.gov)
Description	This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye. (Show More)
Development Status	Active
Indication	Retinitis Pigmentosa
Disease Ontology Term	DOID:10584
Compound Name	ZM-02
Compound Description	rAAV-PsCatCh2.0
Sponsor	Zhongmou Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	12
Results Posted	Not Available

Target Gene/Variant	Ch2.0
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV
Editor Type
Dose 1	Undisclosed low dose
Dose 2	Undisclosed high dose
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China