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NCT04437771ENROLLING BY INVITATION

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Study Summary

This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial. After completion of the FANCOLEN-I study, eligible subjects will be followed for a total of 15 years post gene therapy treatment. No investigational drug product will be administered during this study.

Conditions
Fanconi Anemia Complementation Group AFanconi Anemia
Interventions / Drugs
Safety and efficacy assessments
View on ClinicalTrials.gov ↗
Sponsor
RCKT
Rocket Pharmaceuticals
Gene Therapy
C60/100
Study Details
Enrollment9 participants
Primary Completion—
Lead SponsorRocket Pharmaceuticals Inc.
Data Retrieved2026-04-11 04:25:37.504858+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Current status: ENROLLING BY INVITATION.

Enrolled: 9 participants
Sponsor Catalyst Profile

Rocket Pharmaceuticals (RCKT) is the sponsoring company for this trial. BiotechSign currently grades this company C (60/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

RCKT Full Pipeline →All Companies →
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