Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

NCTID	NCT04483440 (View at clinicaltrials.gov)
Description	This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM). (Show More)
Development Status	Inactive
Indication	Choroideremia
Disease Ontology Term	DOID:9821
Compound Name	4D-110
Compound Description	adeno-associated virus serotype 2 capsid variant (4D R100) carrying a transgene encoding a codon-optimized human choroideremia (CHM) gene
Sponsor	4D Molecular Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	13 (ACTUAL)
Results Posted	Not Available

Target Gene/Variant	CHM
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 R100
Editor Type	none
Dose 1	3E11 vg/eye
Dose 2	1E12 vg/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

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