Aspen Neuroscience has initiated Cohort 3 in its ASPIRO Phase 1/2a clinical trial of ANPD001, marking a significant milestone in the development of personalized cell therapy for Parkinson's disease. This cohort represents the first administration of the company's commercial formulation, designed to support scalable manufacturing for future clinical and commercial use.
The biotechnology company announced that the Cohort 3 formulation has been deemed preclinically comparable to formulations used in the first two cohorts, ensuring continuity in the trial progression. Earlier cohorts demonstrated positive six-month data at the International Association of Parkinson's Disease and Related Disorders (IAPRD), showing strong safety and tolerability alongside clinician- and patient-reported improvements, achieved without immunosuppression.
Commercial Formulation Advances Manufacturing Scalability
"Cohort 3 represents an important step toward commercial readiness," said Damien McDevitt, Ph.D., President and CEO of Aspen Neuroscience. "We've optimized our formulation and delivery system to meet the rigorous demands of late-stage development and future market access, while preserving the personalized nature of our autologous approach."
The new commercial formulation allows cryopreserved cells to be available for dosing immediately upon arrival at the institution, streamlining surgical workflows and minimizing impact on hospital cell processing laboratories. This advancement addresses critical scalability challenges that could affect commercial viability.
Three-Pillar Therapeutic Platform
Aspen's approach integrates three proprietary components to deliver ANPD001. The manufacturing platform converts patient-derived skin cells into dopaminergic neuronal precursor cells (DANPCs) using machine learning algorithms to optimize cell quality. The therapeutic platform utilizes autologous induced pluripotent stem cell (iPSC)-derived DANPCs, creating personalized regenerative cell therapy using patients' own cells to avoid immune rejection.
The third component involves a proprietary delivery system combining a metered dosing syringe with MRI guidance to ensure sub-millimeter accuracy during minimally invasive surgery.
"Together, these three pillars form a unified platform that is personalized, precise, and scalable—setting a new standard in autologous iPSC-derived cell therapy," said Lisa Johnson-Pratt, M.D., Executive Vice President, Therapeutic Program Lead.
Addressing Unmet Medical Need
With more than one million people living with Parkinson's disease in the United States alone and no disease-modifying therapies currently available, ANPD001 offers a potential first-in-class opportunity to restore lost dopaminergic function with curative intent.
The autologous approach distinguishes ANPD001 from allogeneic therapies that use donor cells. By using patients' own cells, the therapy avoids immune rejection and eliminates the need for prolonged immunosuppression, reducing associated adverse events and drug-monitoring requirements while enabling treatment for patients with contraindications to immunosuppressive therapies.
Manufacturing Process and Trial Design
Aspen's manufacturing process begins with a small biopsy of the patient's skin cells, followed by reprogramming to induced pluripotent stem cells and differentiation into DANPCs. These cells are transplanted into the posterior putamen to replace cells lost or damaged due to disease. Cell quality is assessed at every manufacturing stage using proprietary machine learning-based genomics tests.
ANPD001 has received Fast Track designation from the U.S. Food and Drug Administration, recognizing its potential to address unmet medical needs.
The ASPIRO trial represents the first multi-patient, multi-center clinical trial of an autologous cell therapy for Parkinson's disease. It evaluates safety, tolerability, and preliminary efficacy of ANPD001 in levodopa-responsive patients aged 50-70. Primary endpoints will be reported at 12 months, with long-term follow-up extending to 15 years.
ANPD001 is positioned as the most advanced autologous investigational cell therapy in the United States for treating Parkinson's disease. The trial is supported by funding from the California Institute for Regenerative Medicine, which enabled the first two cohorts and continues to support the third cohort's advancement.
