Gene therapy AB-1005 being developed to locally increase glial cell line-derived neurotrophic factor (GDNF) levels
Asklepios BioPharmaceutical (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been randomized at the Ohio State University Wexner Medical Center in the Phase 1 REGENERATE MSA-101 clinical trial of AB-1005, a gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P).
This marks a significant milestone in the development of AB-1005 gene therapy, an adeno-associated viral vector encoding for glial cell line-derived neurotrophic factor (AAV2-GDNF) that is delivered to the putamen, and brings this therapeutic one step closer to potentially reaching patients. AB-1005 is also currently being investigated for the treatment of mild to moderate Parkinson’s disease with the enrollment of the Phase 1b study having now been completed.
“It means a lot to the MSA community to know that the first patient has been enrolled in the Phase 1 REGENERATE MSA-101 trial,” said Philip M. Fortier, MA, President and Executive Director, Defeat MSA Alliance. “There is no cure for MSA, and there are currently no treatments to stop or slow the progression of the disease. This makes it especially hard for patients, given the rapid decline many will experience. Today’s milestone hopefully brings us one step closer to potentially changing the outcome for MSA patients.”
MSA-P, which can initially be difficult to distinguish from Parkinson’s disease, is marked by slow movement, lack of coordination, imbalance, and dizziness, among other symptoms, as individuals experience increasing difficulty with movement.
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