Aspen Neuroscience, Inc., a biotechnology company focused on autologous regenerative therapies, has announced the initiation of Cohort 3* in its ASPIRO Phase 1/2a clinical trial for ANPD001, a personalized dopaminergic neuronal precursor cell (DANPC) therapy aimed at treating moderate to advanced Parkinson's disease (PD). This significant milestone marks the first use of Aspen's commercial formulation of ANPD001, which is designed to facilitate scalable and reproducible manufacturing for both clinical and commercial applications. The formulation for Cohort 3 has been shown to be preclinically comparable to those used in the initial two cohorts, ensuring a smooth transition into this next phase. Earlier cohorts reported positive six-month data at IAPRD, which indicated strong safety and tolerability, along with improvements noted by both clinicians and patients, 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 formulation enables the cryopreserved cells to be ready for dosing immediately upon arrival at the institution, enhancing the surgical workflow and reducing the burden on hospital cell processing labs.

Aspen's commercial formulation of ANPD001 introduces a new standard for neurodegenerative disease treatment through a proprietary integrated approach. "Together, these three pillars form a unified platform that is personalized, precise, and scalable—setting a new standard in autologous iPSC-derived cell therapy," stated 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."

ANPD001's autologous approach, which utilizes a patient's own cells, distinguishes it in the PD treatment landscape. Unlike allogeneic therapies that rely on donor cells, ANPD001 mitigates the risk of immune rejection and the associated need for long-term immunosuppression. Its personalized design enables tailored treatment, while the commercial formulation guarantees consistency and scalability. With over one million individuals in the U.S. living with Parkinson’s disease and no current disease-modifying therapies available, ANPD001 presents a potential first-in-class opportunity to restore lost dopaminergic function with the intent of curing the disease.

About ANPD001, it is the most advanced autologous investigational cell therapy in the United States for treating Parkinson's disease. More details regarding the Phase 1/2a trial can be found at clinicaltrials.gov (NCT06344026). Aspen's personalized approach eliminates the need for immunosuppressive drugs, which can lead to adverse events and monitoring requirements, thereby allowing dosing for patients who have contraindications to immunosuppressive therapies. The manufacturing process begins with a small biopsy of the patient's skin cells, which are reprogrammed into induced pluripotent stem cells (iPSCs) and then differentiated into DANPCs. These cells are transplanted into the posterior putamen to replace those lost or damaged due to disease. The quality of each individual's cells is evaluated at every stage of manufacturing utilizing Aspen's proprietary machine learning-based genomics tests. ANPD001 has also received Fast Track designation from the U.S. Food & Drug Administration (FDA).

About the ASPIRO Trial, it is the first multi-patient, multi-center clinical trial evaluating an autologous cell therapy for Parkinson's disease, focusing on the safety, tolerability, and preliminary efficacy of ANPD001 in patients aged 50–70 who are responsive to levodopa. Primary endpoints are set to be reported at 12 months, with long-term follow-up extending up to 15 years. For more information, please visit www.aspenneuroscience.com. *We are entering testing for the third Cohort, building on funding provided by the California Institute for Regenerative Medicine (CIRM), a state of California agency that supports regenerative medicine, stem cell, and gene therapy research, which enabled Cohorts 1 and 2.