Aspen Neuroscience has initiated Cohort 3 in its ASPIRO Phase 1/2a clinical trial of ANPD001, a personalized dopaminergic neuronal precursor cell (DANPC) therapy for patients with moderate to advanced Parkinson’s disease (PD).
This marks the first use of Aspen’s commercial formulation of ANPD001, designed for scalable and reproducible manufacturing to support future clinical development and potential commercialization. The company reports that preclinical testing found the Cohort 3 formulation comparable to those used in earlier cohorts.
In previous cohorts, six-month data presented at the International Association of Parkinsonism and Related Disorders (IAPRD) meeting indicated strong safety and tolerability, as well as clinician- and patient-reported improvements – all achieved without immunosuppression.
“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 ready for dosing immediately upon arrival at clinical sites. According to Aspen Neuroscience, this streamlines surgical workflow and reduces demands on hospital-based cell processing laboratories.
Aspen’s Approach: Three Pillars
- Manufacturing Platform: Patient-derived skin cells are converted into DANPCs using machine learning algorithms intended to optimize cell quality.
- Therapeutic Platform: ANPD001 is an autologous induced pluripotent stem cell (iPSC)-derived DANPC therapy that uses a patient’s own cells, aiming to avoid immune rejection by not requiring immunosuppressive drugs.
- Proprietary Device: The company employs a precision delivery system combining metered dosing syringes with MRI guidance for 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. “This commercial formulation is designed not just for clinical success, but for real-world impact. Enabling precision dosing without immunosuppression, ensuring scalable and consistent cell quality, and simplifying surgical workflows reduces the time and burden on hospital cell processing labs which is critical for commercial viability. Our vision is to deliver a commercial-ready solution positioned to redefine how neurodegenerative diseases are treated.”
Aspen reports that its autologous approach positions ANPD001 distinctly among PD therapies by avoiding immune rejection issues seen with allogeneic products (which use donor cells). The aim is tailored treatment without prolonged need for immunosuppression while maintaining manufacturing consistency at scale.
The company notes there are over one million people living with Parkinson’s disease in the U.S., with no currently available disease-modifying therapies. According to Aspen Neuroscience, ANPD001 may represent an opportunity as a first-in-class therapeutic aimed at restoring lost dopaminergic function.
About ASPIRO Trial
- This trial tests safety, tolerability, and early efficacy outcomes in levodopa-responsive PD patients aged 50–70 across multiple sites.
- Main endpoints will be assessed after twelve months; long-term follow-up will continue up through fifteen years post-treatment.
- Cohorts 1 & 2 were funded through grants provided by California Institute for Regenerative Medicine (CIRM).
- More information can be found on here – NCT06344026.
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