A Study of AB-1005 Gene Therapy in Japanese Adults With Moderate Parkinson's Disease (J-REGENERATE-PD)

A Phase 2, Open-label, Single Group, Fixed-dose Study to Evaluate the Safety and Efficacy of a Single Bilateral Intraputaminal Administration of AB-1005 (AAV2-GDNF Gene Therapy) in Japanese Participants With Moderate Stage Parkinson's Disease

The objective of this Phase 2, open-label, single group, one-time, fixed-dose study is to assess the safety and efficacy of bilateral intraputaminal infusion of AB-1005 (AAV2-GDNF gene therapy) in Japanese patients with moderate Parkinson's Disease (PD).

Inclusion Criteria:

• Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent

• Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) and currently meeting all the following criteria:

  1. Presence of bradykinesia PLUS any of the following: rigidity, rest tremor, postural instability
  2. Presence of motor fluctuations as measured by the PD Motor Diary
  3. Stable anti-parkinsonian medication regimen for 4 weeks or more, prior to screening
  4. Responsiveness to levodopa therapy

Exclusion Criteria:

• Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to idiopathic PD diagnosis
• Presence or history of significant vascular and/or cardiovascular disease
• Presence of clinically significant cognitive impairment
• Presence or history of psychosis or impulse control disorder
• History of malignancy other than treated cutaneous squamous or basal cell carcinomas
• Presence of clinically relevant conditions that could compromise surgical suitability and/or subject safety
• Contraindication to magnetic resonance imaging and/or use gadolinium-based contrast agents
• Prior history of brain surgery including, but no limited: DBS, pallidotomy focused ultrasound thalamotomy, or other experimental neurosurgical procedure
• Chronic immunosuppressive therapy