Recent Updates
Recently added Catalysts

AAV2-GDNF gene therapy

Phase 2

Parkinson Disease | Small molecule | Neurology |Bayer AG|Last Updated: Jun 29, 2026

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindSHAM_CONTROLLEDDMC
Total Trials1
Total Enrollment127

FDA Designations

No designations recorded

Clinical trial landscape

AAV2-GDNF gene therapy · 1 trial · 1 indication

Phase 2 1
NCT06285643A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)Parkinson Disease
RECRUITING127 Analytics
PHASE2RECRUITING
A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
Parkinson DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from baseline to Month 18 on PD Motor Diary
18 months

Normalized Good ON time per Hauser Diary

Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AAV2-GDNFEXPERIMENTAL -
Control SurgerySHAM_COMPARATOR -

Interventions

NameTypeDescription
AAV2-GDNF gene therapyDRUGBilateral image-guided infusion of AAV2-GDNF into putamen, single dose
control surgeryPROCEDUREBilateral partial burr/twist holes without dural penetration
Unlock Study Design Details

Eligibility Criteria

Age Range45 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites46

Inclusion Criteria: Age 1. Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent Type of Subject and Disease Characteristics 2. Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) as defined by the following: 1. Presence of bradykines...

Countries:United StatesGermanyPolandUnited Kingdom
Unlock Eligibility Criteria

Frequently asked questions about AAV2-GDNF gene therapy

What is AAV2-GDNF gene therapy used for in Parkinson Disease?

AAV2-GDNF gene therapy is an investigational treatment for moderate Parkinson Disease. It is designed to deliver the GDNF gene to the brain to potentially restore or protect dopamine-producing neurons. The therapy is currently being evaluated in a Phase 2 clinical trial for adults with moderate Parkinson Disease.

What does AAV2-GDNF gene therapy target?

AAV2-GDNF gene therapy targets the delivery of the GDNF (glial cell line-derived neurotrophic factor) gene to the brain. This gene encodes a protein that supports the survival and function of dopamine-producing neurons, which are progressively lost in Parkinson Disease. The therapy aims to slow or halt disease progression.

Who makes AAV2-GDNF gene therapy?

AAV2-GDNF gene therapy is being developed by Bayer AG, a multinational pharmaceutical company. Bayer's stock is traded under the ticker symbol BAYRY on the OTC market. The company is conducting the REGENERATE-PD trial to evaluate this therapy for Parkinson Disease.

What phase is AAV2-GDNF gene therapy in?

AAV2-GDNF gene therapy is in Phase 2 clinical development. It is an investigational therapy, meaning it has not been approved by regulatory authorities. The ongoing Phase 2 trial, REGENERATE-PD, is recruiting participants to assess the safety and efficacy of this treatment for moderate Parkinson Disease.

What clinical trials is AAV2-GDNF gene therapy in?

AAV2-GDNF gene therapy is being studied in the REGENERATE-PD trial, identified by the clinical trial number NCT06285643. This Phase 2, randomized, double-blind, sham-controlled study is recruiting 127 adults with moderate Parkinson Disease. The trial is being conducted in the United States, Germany, Poland, and the United Kingdom.

Is AAV2-GDNF gene therapy the same as GDNF gene therapy?

AAV2-GDNF gene therapy is a specific form of GDNF gene therapy. The name 'AAV2-GDNF' indicates that the GDNF gene is delivered using an adeno-associated virus serotype 2 (AAV2) vector. This distinguishes it from other GDNF delivery methods, such as direct protein infusion or different viral vectors.