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FDA Grants Approval for Investigational New Drug Application by Aspen Neuroscience ANPD001, an Autologous Cell Therapy for Parkinson's Disease

Wednesday, August 09, 2023

Aspen Neuroscience has revealed that the U.S. Food and Drug Administration (FDA) has given the green light to its Investigational New Drug (IND) application. This development signifies a significant step forward, allowing the company to move ahead with a clinical trial centered around ANPD001. ANPD001 serves as a personalized (autologous) cell therapy geared towards treating Parkinson's Disease by replenishing the lost dopamine neurons.

The subsequent phase in Aspen's plan involves the commencement of a Phase 1/2a clinical trial, intended for individuals grappling with moderate to severe Parkinson's Disease (PD). This initiative comes on the heels of the company's Trial-Ready Screening Cohort Study in 2022. This prior study's primary aim was to identify suitable candidates, enroll them, and initiate the manufacturing of cells tailored to potential participants for the impending clinical trial. An interesting facet of this endeavor is that it will mark the inaugural multicenter Phase 1/2a trial of an autologous iPSC-derived therapy conducted within the United States.

Aspen Neuroscience has introduced a novel approach involving induced pluripotent stem cells (iPSCs), which are derived from a patient's own skin cells. These iPSCs are utilized to create dopamine neuronal precursor cells (DANPCs), a crucial element of the therapy. Rigorous quality control measures, including Aspen's unique artificial intelligence-driven genomics tests, are applied to evaluate the viability of these cells before transplantation.

Faheem Hasnain, Chairman of the Aspen board of directors, hailed this achievement as a significant advancement in their mission to develop personalized iPSC-derived cell replacement therapies, beginning with Parkinson's disease. He emphasized the collaborative efforts of the Aspen team and the invaluable contribution of the patients in propelling the company's progress.

Damien McDevitt, PhD, Aspen's President and CEO, described the FDA's clearance of the Investigational New Drug (IND) application for ANPD001 as a momentous step towards addressing the needs of the millions of people worldwide affected by Parkinson's disease. He expressed optimism about the potential of personalized regenerative medicine and the prospect of bringing relief to patients through this cell therapy.

The groundwork for Aspen's autologous cell therapy technique was laid by co-founders Jeanne Loring, PhD, and Andres Bratt-Leal, PhD. Loring, a Professor Emeritus and Director of The Center for Regenerative Medicine at the Scripps Research Institute, and Bratt-Leal, Aspen's Senior Vice President of Research and Development, pioneered this innovative approach.

ANPD001, the investigational cell therapy, aims to replace neurons in Parkinson's disease patients. The multi-stage manufacturing process involves obtaining a small skin sample from the patient, reprogramming it into iPSCs, and further differentiating them into DANPCs. These DANPCs are then surgically implanted to replace the damaged or lost neurons. The quality of the cells is vigilantly monitored using Aspen's proprietary AI-based genomics tests throughout the manufacturing phases.

The Phase 1/2a Clinical Trial for ANPD001 is designed as a single-arm, open-label study to assess its safety, tolerability, and preliminary effectiveness for patients dealing with moderate to severe Parkinson's disease. The study includes a planned dose escalation to explore the therapeutic potential of the treatment.

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