AskBio adds 2 neurodegenerative gene therapy programs to pipeline

By The Science Advisory Board staff writers

January 28, 2021 -- The clinical team from Brain Neurotherapy Bio, an Ohio-based gene therapy company, has joined Asklepios BioPharmaceutical (AskBio), a Bayer company, to expand its clinical pipeline for the treatment of neurodegenerative disorders.

Leaders from Brain Neurotherapy Bio will lead AskBio's development efforts in Parkinson's disease and multiple system atrophy. Both programs utilize an adeno-associated viral (AAV) vector platform. The product candidate for Parkinson's disease and multiple system atrophy will deliver the glial cell-derived neurotrophic factor (GDNF) gene therapy investigational agent via a novel, image-guided neurosurgical delivery technology in an effort to optimize the precision, safety, and efficiency of gene transfer within the brain.

Brain Neurotherapy's lead clinical program, GDNF gene therapy for Parkinson's disease, is currently in phase IB clinical trials in the U.S. and has treated seven out of 12 patients. The one-time treatment involves infusion of a gene therapy solution into deep structures of the brain that are affected by Parkinson's.

A phase I/II study with a multiple system atrophy program, which has an active investigation new drug application, is expected to begin enrollment in early 2021.

Financial details of the collaboration were not disclosed.

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