Translational Candidate

autologous iPSC-derived dopaminergic progenitor cells

Area of Impact

Parkinson's Disease

Mechanism of Action

Autologous iPSC-derived dopaminergic progenitor cells represent a promising strategy to replace the nigrostriatal cells which are lost in Parkinson's Disease (PD). While approaches using fetal tissue / allogeneic stem cells show great promise, they are not sufficiently personalized to provide maximal safety and efficacy to the broadest demographic of PD patients. If successful, this cell replacement therapy could dramatically improve the standard of care and prognosis for PD patients.

Unmet Medical Need

Current medicines for PD only address the symptoms of the disease by boosting dopamine production from nigrostriatal neurons which continue to degenerate. The proposed approach will replace the degenerating neurons and in this way slow, halt, or even reverse the progression of the disease.

Project Objective

Pre-IND meeting; GMP-ready processes

Major Proposed Activities

• collect tissue samples, generate iPSC, and produce dopaminergic neurons from several PD patient and healthy volunteer donors
• assess the reliability and iteratively improve processes for manufacture and QC of autologous replacement cells to apply to all PD patients
• perform animal studies and complete the data package to submit to the FDA for a Pre-IND meeting

Currently Parkinson’s Disease afflicts approximately 100,000 Californians, exacting tremendous economic and emotional tolls on individuals and society. A disease-modifying personalized cell replacement therapy for PD would improve this situation dramatically, for our families, and at a socioeconomic health system level. The state may save hundreds of millions of dollars in healthcare costs per year.