Asklepios BioPharmaceutical (AskBio) has randomised the first subject in the Phase I REGENERATE MSA-101 clinical trial of gene therapy AB-1005 (AAV2-GDNF) to potentially treat multiple system atrophy-parkinsonian type (MSA-P).

This double-blind, randomised, placebo-controlled trial will analyse the safety of AB-1005 delivered to the putamen in adult MSA-P patients aged 35 to 75 years. 

It will enrol up to nine subjects who will be randomised on the day of the surgical procedure to receive either the gene therapy or a control surgery. 

The first patient was randomised at the Ohio State University Wexner Medical Center in the US.

Furthermore, subject enrolment is underway at the University of California Irvine and the Quest Research Institute, Michigan. 

AskBio subsidiary Brain Neurotherapy Bio is sponsoring this Phase I trial. 

An adeno-associated viral vector that encodes for glial cell line-derived neurotrophic factor, the gene therapy is being analysed to treat mild to moderate Parkinson’s disease.

The company has concluded subject enrolment in a Phase Ib clinical trial for Parkinson’s.

AskBio Parkinson’s and MSA scientific chair Krystof Bankiewicz said: “The clinical advancement of AB-1005 for the treatment of MSA-P comes as we are gathering the results of our Phase I programmes in Parkinson’s. 

“Taken together we expect the outcomes of these trials to meaningfully contribute to the body of scientific knowledge related to the potential of GDNF gene therapy in the treatment of these debilitating central nervous system diseases. 

“We believe these data, combined with our exploration of AAV-based intracranial gene delivery and our close collaborations with clinical neurosurgical centres of excellence, will support our goal of successfully developing and eventually delivering to patients these much-needed treatments.”

In August this year, the company dosed the first subject in the Phase I/II LION-CS101 trial of AB-1003 gene replacement therapy for treating limb-girdle muscular dystrophy type 2I/R9.