More than one million people in the U.S. live with Parkinson’s disease, a condition with no available disease-modifying therapies.
A new development from Aspen Neuroscience offers a potential new direction, as the company initiates a new phase in its clinical trial for a personalized cell therapy.
ASPIRO Trial Enters Third Cohort
Aspen Neuroscience has announced the start of Cohort 3 in its ASPIRO Phase 1/2a clinical trial. This stage is significant because it marks the first time the company’s commercial formulation of ANPD001 is being administered to patients.
This new formulation is designed for scalable and reproducible manufacturing, a critical step for future clinical and commercial use.
The company confirmed that this version is preclinically comparable to the one used in the first two cohorts, which showed strong safety and positive patient-reported improvements without the need for immunosuppression.
What is ANPD001?
ANPD001 is a personalized cell therapy for individuals with moderate to advanced Parkinson’s disease. It is the most advanced autologous investigational cell therapy for this condition in the United States.
The therapy uses a patient’s own skin cells, which are reprogrammed into dopaminergic neuronal precursor cells (DANPCs). These cells are then transplanted back into the patient to replace those lost or damaged by the disease.
| Feature | Aspen’s Autologous Approach (ANPD001) | Allogeneic Therapy |
|---|---|---|
| Cell Source | Patient’s own cells | Foreign donor cells |
| Immune Rejection | Avoided | Potential for rejection |
| Immunosuppression | Not required | Prolonged immunosuppression needed |
| Treatment | Personalized and customized | Standardized |
“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.”
Three Pillars of Innovation
Aspen’s approach is built on three integrated components to ensure a personalized, precise, and scalable treatment.
- Manufacturing Platform: A proprietary process converts a patient’s skin cells into high-quality DANPCs, using machine learning to ensure cell quality.
- Therapeutic Platform: The use of a patient’s own cells in ANPD001 avoids immune rejection and the need for immunosuppressive drugs.
- Proprietary Delivery Device: A precision delivery system uses MRI guidance and a metered dosing syringe to ensure sub-millimeter accuracy during the minimally invasive surgery.
The ASPIRO Trial Explained
The ASPIRO trial is the first multi-center clinical study of an autologous cell therapy for Parkinson’s disease. It evaluates the safety, tolerability, and preliminary efficacy of ANPD001.
The trial enrolls levodopa-responsive patients between the ages of 50 and 70. Primary results will be reported at 12 months, with a long-term follow-up planned for 15 years.
ANPD001 has received Fast Track designation from the U.S. Food & Drug Administration (FDA). More information about the Phase 1/2a trial is available at clinicaltrials.gov under identifier NCT06344026.
A New Horizon for Parkinson’s Treatment
With no current therapies that can modify the course of Parkinson’s disease, ANPD001 represents a potential first-in-class opportunity. By aiming to restore lost dopaminergic function, this personalized therapy offers a new possibility for patients.
The initiation of Cohort 3 with a commercial-grade formulation marks a key milestone in bringing this innovative treatment closer to real-world application.
For more information, visit: https://aspenneuroscience.com/
