Actus will benefit from AskBio’s ability to generate organ and tissue-specific single-stranded and self-complementary genome containing adeno-associated virus vectors (AAVs), the company’s manufacturing platform and managements’ experience progressing AAV gene therapy programs into the clinic. Actus will pursue therapies for multiple rare genetic diseases, and will initially focus on developing a gene therapy for Pompe Disease based on the research of Dwight Koeberl, M.D., Ph.D., professor of pediatrics and a medical genetics specialist at Duke University.
“AskBio is focused on expanding our current gene therapy portfolio to help patients and families with rare genetic diseases that might be treated through our proprietary gene-therapy platform,” said Sheila Mikhail, President and CEO of AskBio and Actus.
“We continue to advance the efficacy of our vectors as well as our manufacturing platform, and we are excited to be working with Dr. Dwight Koeberl and his team at Duke, with the aim of developing a new therapy for Pompe Disease.”
Actus will create targeted low-dose gene therapies for orphan diseases using AskBio’s AAV platform technologies and manufacturing process. AskBio’s AAVs are effective at lower doses, have increased cell-type specificity and have the potential for reduced systemic side-effects.
AskBio’s proprietary manufacturing technology has been proven to be more efficient than other manufacturing technologies and has been used in clinical-grade GMP and preclinical-grade GLP manufacturing campaigns as well as several thousand research-grade lots.
“We have shown in mice that AAV gene therapies have the potential to overcome the limitations of enzyme replacement therapy by delivering low doses and restricting expression to the liver,” said Dr. Dwight Koeberl, “We look forward to working with Actus and bringing these therapies into human clinical trials. We are currently recruiting patients for an FDA-approved Phase I trial and hope to be underway early next year.”
Actus is currently recruiting 20 adult patients with late-onset Pompe Disease for the clinical trial.
Pompe Disease is a genetic disease caused by the deficiency of acid-alpha-glucosidase (GAA) in muscle, resulting in the accumulation of glycogen in organs and tissues, especially muscles, which can impair their ability to function normally. While enzyme replacement has shown promise in infantile-onset Pompe patients, no curative therapy is available.