Parkinson’s Disease is a neurodegenerative disease caused by loss of dopaminergic (DA) neurons in the nigrostriatal pathway that leads to progressive loss of motor and neurological function. It is the second most common neurodegenerative movement disorder (after Alzheimer’s), affecting 1 million people in the U.S. alone. PD symptoms (impairments in movement, coordination, speech, and cognition) severely affect quality of life and patient life span. Current therapies are not curative, decrease in effectiveness over time, and can have side effects that impact patient quality of life. Since motor symptom severity correlates with the loss of DA neurons, cell replacement therapy holds great promise for restoring brain function.

ANPD001 is a novel, personalized, regenerative cell therapy. It is composed of dopaminergic neuron precursor cells (DANPCs) derived from autologous induced pluripotent stem cells (iPSCs) from the skin biopsies of PD patients. Aspen’s preclinical data shows that DANPCs transplanted into animal models differentiate into DA neurons that integrate into the brain and halt of reverse PD symptoms. This year ANPD001 received FDA IND clearance in August and Fast Track Designation in October. In 2022, Aspen established an interventional trial-ready cohort screening study, and screened and recruited people with mid-stage to moderately advanced sporadic PD, and collected skin biopsies. We are now generating iPSCs from these biopsies and manufacturing clinical-grade ANPD001. To support the first cohort, ANPD001 for three (3) patients has been manufactured.

More information about the Phase 1/2a study will be announced soon.