ArticleNeurology2022
Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial.
Registry-linked trialOpen access · hybridAbstract read
In one paragraph
Article in Neurology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01973543 (An Open-label Safety and Efficacy Study of VY-AADC01 Administered by MRI-Guided Convective Infusion Into the Putamen of Subjects With Parkinson's Disease With Fluctuating Responses to Levodopa), which is not on this map. Cited by 39 papers.
0numbers the graph read from it
0cells of the map it votes in
39citing papers in PubMed
5.7field-weighted citation impact, top 3% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-label Safety and Efficacy Study of VY-AADC01 Administered by MRI-Guided Convective Infusion Into the Putamen of Subjects With Parkinson's Disease With Fluctuating Responses to Levodopa
Who cites it
39 citing papers in PubMed, 76 citations in OpenAlex.
- AAV2 capsid clearance and neuronal trafficking dynamics in the central nervous system.Gene therapy · 2026Article
- Attenuating AAV-triggered innate immunity in the adult mouse nervous systemActa pharmaceutica Sinica. B · 2026Article
- Dual-target gene therapy in Parkinson's disease: a multicenter phase 1 trial.Nature medicine · 2026Article
- Clinical GDNF delivery methods for Parkinson's disease.Journal of Parkinson's disease · 2026Review
- Advancing AADC deficiency therapy through MR-guided multisite delivery of AAV2-hAADC to dopaminergic and serotonergic pathways in the brainstem.Molecular therapy. Advances · 2026Article
- Involvement of Neuroinflammation and Oxidative Stress in L-DOPA-Induced Dyskinesia in Parkinson's Disease: Role of Renin-Angiotensin System and ROCK Pathway.Antioxidants (Basel, Switzerland) · 2025Review
- Gene therapy downregulating CaV1.3 reverses motor disability in aged parkinsonian male and female macaques.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Investigational Gene Therapies for Parkinson's Disease.CNS drugs · 2025Review
- Optimized AAV capsids for basal ganglia diseases show robust potency and distribution.Nature communications · 2025Article
- Two-year feasibility and safety of open-label autologous peripheral nerve tissue implantation during deep brain stimulation in patients with Parkinson's disease.Journal of Parkinson's disease · 2025Article
- Genetic therapies for movement disorders - current status.Journal of neurology · 2025Review
- Viruses and neurodegeneration: a growing concern.Journal of translational medicine · 2025Review
- Significance of gene therapy in neurodegenerative diseases.Frontiers in neuroscience · 2025Review
- Viral vectors in neurodegenerative diseases: immune responses and therapeutic applications.Frontiers in neurology · 2025Review
- Review
- Gene Therapy for Parkinson's Disease Using Midbrain Developmental Genes to Regulate Dopaminergic Neuronal Maintenance.International journal of molecular sciences · 2024Review
- A review of temporal interference, nanoparticles, ultrasound, gene therapy, and designer receptors for Parkinson disease.NPJ Parkinson's disease · 2024Review
- From lab bench to hope: a review of gene therapies in clinical trials for Parkinson's disease and challenges.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024Review
- Advancements in surgical treatments for Huntington disease: From pallidotomy to experimental therapies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024Review
- Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside.Journal of translational medicine · 2024Review
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Authors and funding
14 authors at 1 institution in 1 country.
Chadwick W ChristineFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus. chad.christine@ucsf.edu.
R Mark RichardsonFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.ORCID 0000-0003-2620-7387
Amber D Van LaarFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.ORCID 0000-0002-8912-3159
Marin E ThompsonFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Elisabeth M FineFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Omar S KhwajaFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Chunming LiFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Grace S LiangFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Andreas MeierFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Eiry W RobertsFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Madeline L PfauFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Josh R RodmanFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Krystof S BankiewiczFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Paul S LarsonFrom the Departments of Neurology (C.W.C.) and Neurological Surgery (M.E.T., K.S.B., P.S.L.), University of California, San Francisco; Department of Neurosurgery (R.M.R.), Massachusetts General Hospital; Harvard Medical School (R.M.R.), Boston, MA; Department of Neurology (A.D.V.L.), University of Pittsburgh Medical Center, PA; Brain Neurotherapy Bio (A.D.V.L.), Inc., Columbus, OH; Voyager Therapeutics, Inc. (E.M.F., O.S.K., C.L., A.M.), Cambridge, MA; Neurocrine Biosciences, Inc. (G.S.L., E.W.R.), San Diego, CA; ApotheCom (M.L.P., J.R.R.), New York, NY; and Department of Neurological Surgery (K.S.B.), Ohio State University, Columbus.
Harvard University · US
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectivesTo 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.
methodsPD-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 × 10
resultsNo serious adverse events (SAEs) attributed to VY-AADC01 were reported. All 4 non-vector-related SAEs (atrial fibrillation and pulmonary embolism in 1 participant and 2 events of small bowel obstruction in another participant) resolved. Requirements for PD medications were reduced by 21%-30% in the 2 highest dose cohorts at 36 months. Standard measures of motor function (PD diary, Unified Parkinson's Disease Rating Scale III "off"-medication and "on"-medication scores), global impressions of improvement (Clinical Global Impression of Improvement, Patient Global Impression of Improvement), and quality of life (39-item Parkinson's Disease Questionnaire) were stable or improved compared with baseline at 12, 24, and 36 months following VY-AADC01 administration across cohorts. DISCUSSIONS: VY-AADC01 and the surgical administration procedure were well-tolerated and resulted in stable or improved motor function and quality of life across cohorts, as well as reduced PD medication requirements in cohorts 2 and 3 over 3 years. TRIAL REGISTRATION INFORMATION: NCT01973543. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that, in patients with moderately advanced PD and motor fluctuations, putaminal infusion of VY-AADC01 is well tolerated and may improve motor function.
Indexed as
Carboxy-LyasesParkinson DiseaseAmino AcidsAntiparkinson AgentsGenetic TherapyHumansQuality of LifeTreatment OutcomeAmino AcidsAntiparkinson AgentsCarboxy-Lyases
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Registered trials
NCT01973543ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.