Gene therapy for Parkinson’s disease progresses to Phase II

Cell and gene therapies

A new Phase II trial, REGENERATE-PD, will study AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease.

The trial will include an estimated 87 subjects with study sites located in the US, UK and Europe.

In January, AskBio announced that the Phase Ib trial of AB-1005 met its primary endpoint, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen.

The investigational gene therapy for the treatment of Parkinson’s disease was well tolerated with no serious adverse events that were considered related to AB-1005 in all 11 patients at 18 months.

“By enhancing levels of a naturally occurring growth factor, glial cell line-derived neurotrophic factor (GDNF) gene therapy is intended to promote the survival and functioning of vulnerable brain cells that degenerate in Parkinson’s disease,” said Alan Whone, Consultant Senior Lecturer in Movement Disorder, Bristol Medical School, and Honorary Consultant Neurologist at North Bristol Trust, UK.

“The advancement of AB-1005 is a significant milestone in the development of a gene therapy for Parkinson’s disease and has the potential to bring an effective treatment one step closer to patients.”

A “transformative impact” in Parkinson’s

AskBio is also exploring GDNF therapy beyond Parkinson’s disease and is currently enrolling patients in the US with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase I trial.

“Following the encouraging results from the Phase Ib study and the presentation of 18-month data at the American Association of Neurology (AAN) meeting in April, we are excited to be progressing AB-1005 to this larger, Phase II study,” said Krystof Bankiewicz, Scientific Chair, Parkinson’s and MSA, AskBio. “This latest advancement highlights our confidence in the potential of AB-1005 to provide a transformative impact for patients with Parkinson’s disease.”

Diana Spencer, Senior Digital Content Editor, DDW

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