Drug intelligence / Profile preview

ANPD002

Development stage
Preclinical
Lead developer
Aspen Neuroscience
Modality
Patient-derived iPSCs → iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies
Administration
Intracranial
01

Overview

ANPD002 is an investigational, gene-edited autologous induced pluripotent stem cell (iPSC)-derived dopaminergic neuron replacement therapy being developed by Aspen Neuroscience for genetic forms of Parkinson’s disease, particularly GBA-associated Parkinson’s disease.[5][9][11][13][15] Starting from a patient’s own somatic cells, Aspen applies iPSC reprogramming and targeted gene correction to repair disease-causing mutations, then differentiates the corrected cells into dopaminergic neuronal precursors for stereotactic implantation into the striatum, with the goal of restoring physiological dopamine signaling and modifying disease progression in patients with monogenic PD.[5][9][11][13][15] This personalized, autologous approach is designed to avoid chronic immunosuppression while directly addressing both the underlying genetic defect and the loss of nigrostriatal dopaminergic neurons that characterize genetic Parkinson’s disease.[5][9][11][13][15]

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