Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

NCTID	NCT05222178 (View at clinicaltrials.gov)
Description	This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management. (Show More)
Development Status	Inactive
Indication	Phenylketonuria (PKU)
Disease Ontology Term	DOID:9281
Compound Name	HMI-103
Sponsor	Homology Medicines, Inc
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	3
Results Posted	Not Available

Target Gene/Variant	PAH
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Hepatocyte
Delivery System	Viral transduction
Vector Type	AAVHSC15
Editor Type
Dose 1	6E13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
FDA Designations	Fast Track
Recent Updates	Homology Medicines has discontinued development of HMI-103