AAV Gene Therapy Study for Subjects with PKU

NCTID	NCT04480567 (View at clinicaltrials.gov)
Description	This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy. (Show More)
Development Status	Inactive
Indication	Phenylketonuria (PKU)
Disease Ontology Term	DOID:9281
Compound Name	BMN 307
Sponsor	BioMarin Pharmaceutical
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	100
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
Delivery System	Viral transduction
Vector Type	AAV5
Editor Type
Dose 1	Dose escalation with 3 levels, unknown concentrations
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States,United Kingdom

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation
Recent Updates	BioMarin (developer of pegvaliase) has made no official announcement of discontinuation. Clinical hold was placed when mice in preclinical studies developed tumors, and Biomarin was sued on allegations that Biomarin had overstated BMN 307's clinical and commercial prospects.