Drug intelligence / Profile preview

ANDP002

Development stage
Preclinical
Lead developer
Aspen Neuroscience
Modality
Cell Therapies
Administration
Intracranial
01

Overview

**ANDP002** is an investigational autologous gene-corrected cell therapy developed by Aspen Neuroscience for genetic Parkinson's disease, specifically targeting patients with GBA mutations (about 8% of PD cases). It employs a personalized approach using induced pluripotent stem cells (iPSCs) derived from the patient's own skin cells, which are genetically corrected to address the GBA mutation before differentiation into dopamine neuronal precursor cells (DANPCs). These cells are surgically implanted to replace lost dopamine-producing neurons in the brain, aiming to restore function without requiring immunosuppression. As of 2022 plans, it is positioned as the company's second PD candidate following ANPD001, with clinic entry targeted by 2024.[11][12]

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