4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

NCTID	NCT04517149 (View at clinicaltrials.gov)
Description	This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR). (Show More)
Development Status	Inactive
Indication	X-Linked Retinitis Pigmentosa
Disease Ontology Term	DOID:0110414
Compound Name	4D-125
Sponsor	4D Molecular Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	21
Results Posted	Not Available

Target Gene/Variant	RPGR
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	AAV
Editor Type	none
Dose 1	3E11 vg/eye
Dose 2	1E12 vg/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	8
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	January 2025: Sponsor announced they would terminate development of this program