AskBio Commences Patient Dosing For Gene Therapy

Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and operated independently as a subsidiary of Bayer AG, announced that the first patient 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), was randomized at the Ohio State University Wexner Medical Center.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, as per pharmabiz.“It means a lot to the MSA community to know that the first patient has been enrolled in the phase 1 MSA-101 trial REGENERATE ,” 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.”Slow movement, poor coordination, imbalance, and dizziness are among the symptoms of MSA-P, which can be hard to differentiate from Parkinson's disease at first. These symptoms worsen as people become more unable to move. This is the outcome of the brain and spinal cord's nerve cells gradually dying off. MSA is an uncommon condition that affects an estimated 100,000–500,000 people globally. It often appears to arise randomly. People in their 50s often start to have symptoms, which quickly develop over the next five to ten years.