The first European patients have been randomised in a Phase II gene therapy clinical trial for Parkinson’s disease (PD), AskBio has announced.  

The REGENERATE-PD clinical trial is evaluating the safety and efficacy of AskBio’s investigational gene therapy AB-1005 and will enrol approximately 87 patients across clinical trial centres in Germany, Poland, the United Kingdom and the United States.  

PD is a progressive, neurodegenerative disorder affecting 1.2 million people in Europe – a number expected to double by 2030, according to the European Parkinson’s Disease Association (EPDA).  

AB-1005 is a gene therapy based on the adeno-associated viral vector serotype 2 (AAV2) and which contains the human glial cell line-derived neurotrophic factor (GDNF) transgene. AskBio states that this allows for stable and continuous expression of GDNF in localised regions of the brain following direct neurosurgical injection.  

Through the REGENERATE-PD clinical trial, AskBio will test the GDNF hypothesis that it can promote the survival and morphological differentiation of dopaminergic neurons. The aim is to get GDNF to degenerating nigrostriatal neurons in patients in a more clinically relevant manner. 

Earlier this year, AB-1005 was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA), meeting the requirements for a gene therapy intended to slow disease progression and improve motor outcomes in patients with PD. RMAT provides recipients with enhanced access to the FDA, which could include intensive guidance on efficient drug development, rolling Biologics License Application (BLA) review, and other actions to expedite review. 

“I believe the randomisation of the first European participants in REGENERATE-PD, which makes this the first neurosurgical gene therapy program for Parkinson’s to successfully randomise patients from both the United States and Europe into a single Phase II trial, is positive news for people living with Parkinson’s disease and the physicians treating them,” said Alan Whone, REGENERATE-PD Europe Lead and Principal Investigator. “There is a significant need for neurorestorative therapies in Parkinson’s and seeing the advancement of an important investigational gene therapy in a Phase II clinical trial will give hope to patients and the medical community alike.”