Aspen Neuroscience Publishes Preclinical Data on Parkinson’s iPSC-Derived Cell Therapy
Researchers used whole-genome sequencing and a machine-learning RNA assay to assess dopamine neuron precursors made from patients’ skin cells for identity and consistency.
Key Points
- Aspen Neuroscience published peer-reviewed preclinical data supporting sasineprocel, an investigational autologous (sourced from the patient) cell therapy for Parkinson’s disease.
- Researchers used whole-genome sequencing and NeuriTest, a machine-learning RNA sequencing assay, to assess cell identity, consistency and preclinical performance.
- Preclinical models showed improved motor function, while a nine-month rodent safety study reported no treatment-related adverse findings.
Aspen Neuroscience has announced the publication of “Autologous iPSC-Derived Cell Therapy for Parkinson’s Disease: Preclinical Evaluation through Genomic Quality Controls” in Cell Stem Cell.
The peer-reviewed paper describes the scientific and translational foundation for sasineprocel (ANPD001), an investigational autologous (sourced from the patient) induced pluripotent stem cell (iPSC)-derived dopamine neuron replacement therapy for Parkinson’s disease. The therapy is designed to restore dopamine and neural circuitry lost during disease progression.
“As we enter Phase 3, it is an honor to share the early research that formed the strong foundation for the high-tech and high-quality development of sasineprocel translating personalized cell therapy from scientific promise into clinical development,” said Xiaokui Zhang, Ph.D., Chief Scientific Officer of Aspen Neuroscience. “This work shows how we integrated whole-genome sequencing and transcriptomic analyses with functional preclinical studies to inform product quality. NeuriTest™ is an important part of that framework and illustrates how data-driven, machine learning tools can support the translation of precise personalized cell therapies from the laboratory to the clinic.”
Genomic and Transcriptomic Quality Controls
The preclinical research evaluated the characterization and manufacturing consistency of dopaminergic neuron precursor cells (DANPCs) produced from multiple patient-specific cell lines. Researchers used whole-genome sequencing data as part of the product quality control framework.
The paper also describes NeuriTest™, Aspen’s RNA sequencing-based bioinformatics assay. NeuriTest applies machine-learning methods to transcriptomic data to determine whether patient-specific DANPC products exhibit molecular characteristics associated with the intended cell identity and preclinical performance. The assay is intended to support consistent product quality across individualized autologous cell therapies.
Preclinical Findings
The reported preclinical findings included:
- Reproducible characterization and manufacturing of DANPCs across multiple cell lines.
- Improved motor function in preclinical Parkinson’s disease models.
- No treatment-related adverse findings in a nine-month Good Laboratory Practice safety study conducted in a rodent model.
These data supported advancing the therapy into the ongoing Phase 1/2a ASPIRO clinical trial. The California Institute for Regenerative Medicine funded the study under grant CLIN2-15547.
Sasineprocel and Its Manufacturing Process
Sasineprocel is a single-dose cell therapy created from a patient’s cells collected through a small skin punch biopsy. The cells are reprogrammed into iPSCs and then differentiated into DANPCs. The resulting cell composition is administered to the putamen using image-guided delivery.
The approach is designed to support the engraftment, survival and functional integration of transplanted cells in the area of the brain where dopamine signaling needs to be restored. Because sasineprocel is autologous (sourced from the patient), the company says immunosuppression required for allogeneic (donor derived) cell therapies is not needed.
Aspen Neuroscience is developing autologous iPSC-derived therapies for neurodegenerative diseases, beginning with Parkinson’s disease. Sasineprocel is its lead product candidate.
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