Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.

ABSTRACT:

Background and objectives

To report final, 36-month safety and clinical outcomes from the PD-1101 trial of NBIb-1817 (VY-AADC01) in participants with moderately advanced Parkinson disease (PD) and motor fluctuations.

Methods

PD-1101 was a phase 1b, open-label, dose escalation trial of VY-AADC01, an experimental AAV2 gene therapy encoding the human aromatic l-amino acid decarboxylase (AADC) enzyme. VY-AADC01 was delivered via bilateral, intraoperative MRI-guided putaminal infusions to 3 cohorts (n = 5 participants per cohort): cohort 1, ≤7.5 × 10¹¹ vector genomes (vg); cohort 2, ≤1.5 × 10¹² vg; cohort 3, ≤4.7 × 10¹² vg.

Results

No serious adverse events (SAEs) attributed to VY-AADC01 were reported. All 4 non-vector-related SAEs (atrial

SUBMITTER: Christine CW 

PROVIDER: S-EPMC8726573 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature