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NCT04348708ENROLLING BY INVITATION

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

Study Summary

An Open-Label, Long-Term Follow Up Study of Safety and Efficacy in PKU Subjects with PAH Deficiency Previously Administered HMI 102

Conditions
PhenylketonuriaPAH Deficiency
Interventions / Drugs
HMI-102
View on ClinicalTrials.gov ↗
Sponsor
FIXX
Homology Medicines
Gene Therapy
D40/100
Study Details
Enrollment21 participants
Primary Completion—
Lead SponsorHomology Medicines, Inc
Data Retrieved2026-04-11 04:14:08.656343+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Current status: ENROLLING BY INVITATION.

Enrolled: 21 participants
Sponsor Catalyst Profile

Homology Medicines (FIXX) is the sponsoring company for this trial. BiotechSign currently grades this company D (40/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

FIXX Full Pipeline →All Companies →
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