Aspen Neuroscience recently announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application, enabling the company to proceed with a clinical trial for ANPD001, which is a personalized (autologous) cell therapy to treat Parkinson’s Disease by replacing lost dopamine neurons.
Aspen now plans to initiate a first-in-patient Phase 1/2a clinical trial for people with moderate to severe Parkinson’s Disease (PD). This follows the company’s 2022 Trial-Ready Screening Cohort Study to screen, enroll, and begin manufacturing cells for potential patient candidates for the clinical trial. And this study will be the first multicenter Phase 1/2a trial of an autologous iPSC-derived therapy in the U.S.
Aspen’s autologous approach uses induced pluripotent stem cells (iPSCs) developed from the patient’s skin cells to manufacture dopamine neuronal precursor cells (DANPCs). And these cells are then evaluated for potential activity using robust quality control assays, including Aspen’s proprietary artificial intelligence-based genomics tests, before being implanted.
The autologous cell therapy approach was pioneered by Aspen’s co-founders Jeanne Loring, Ph.D., Professor Emeritus and Director of The Center for Regenerative Medicine, Department of Molecular Medicine at the Scripps Research Institute, and Andres Bratt-Leal, Ph.D., Aspen senior vice president, research and development.
KEY QUOTES:
“This is a major milestone in Aspen’s mission to develop and deliver personalized iPSC-derived cell replacement therapies for people with unmet medical needs, starting with Parkinson’s disease. This is an exciting time for the Aspen team and the patients who have been so instrumental in enabling the company’s development.”
— Faheem Hasnain, chairman of the Aspen board of directors
“The IND clearance of ANPD001 sets in motion a path toward a new treatment for the more than one million Americans and 10 million people worldwide with Parkinson’s disease. Our visionary team is working to make personalized regenerative medicine a reality, and we look forward to advancing this cell therapy for patients who are waiting.”
— Damien McDevitt, PhD, Aspen president and CEO
