AAV Gene Therapy Study for Subjects with PKU

Study Summary

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.

Conditions

Phenylketonuria (PKU)

Interventions / Drugs

BMN 307

View on ClinicalTrials.gov ↗

Sponsor

BMRN

BioMarin Pharmaceutical

Rare Disease

D36/100

Study Details

Enrollment100 participants

Primary Completion—

Lead SponsorBioMarin Pharmaceutical

Data Retrieved2026-04-11 02:55:23.531553+00:00

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