First Patient Enrolled in AskBio’s Phase I Trial for Multiple System Atrophy-Parkinsonian Type (MSA-P) Gene Therapy
22 November 2023
Gene therapy company, Asklepios BioPharmaceutical, Inc. (AskBio) has announced that the first patient has been randomized at the Ohio State University Wexner Medical Center in the Phase I REGENERATE MSA-101 clinical trial of AB-1005.
The gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P) marks a significant milestone in the development of AB-1005 gene therapy. The gene therapy is an adeno-associated viral vector encoding for glial cell line-derived neurotrophic factor (AAV2-GDNF) delivered to the putamen.
Nicolas M. Phielipp, MD, University of California Irvine, REGENERATE MSA-101 Principal Investigator said:
“We’re including a genetic sequence in the AAV2 vector that codes for the GDNF protein and are delivering this to the putamen. In this way, we’re targeting local brain cells and adjacent brain tissue that can benefit from the protein’s growth properties. This trial marks the first step toward understanding the potential that GDNF gene therapy might have for patients with MSA-P.”
Affecting approximately 100,000–500,000 worldwide, MSA-P can be difficult to differentiate from Parkinson’s disease. MAS-P comprises of dizziness, imbalance, lack of coordination and slow movement, amongst other symptoms. This is the result of a progressive loss of nerve cells in the brain and spinal cord. The rare disease in most cases occurs randomly with most symptoms tending to appear in people during their 50s, followed by a rapid progression within 5–10 years.
Bring this therapeutic one step closer to potentially reaching patients, it’s a significant milestone for the MSA community. Philip M. Fortier, MA, President and Executive Director, Defeat MSA Alliance commented:
“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.”
“The clinical advancement of AB-1005 for the treatment of MSA-P comes as we are gathering the results of our Phase I programs 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 centers of excellence, will support our goal of successfully developing and eventually delivering to patients these much-needed treatments.”
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