A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation
Grant Award Details
Grant Type:
Grant Number:
TRAN1-16012
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$3,999,241
Status:
Pre-Active
Grant Application Details
Application Title:
A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation
Public Abstract:
Translational Candidate
Human pluripotent stem cell expanded and differentiated dopaminergic precursor cells to treat Parkinson’s Disease at high quantity and high quality
Area of Impact
This candidate uses a 3D cell culture technology that addresses the manufacturing bottleneck and creates a high-quality scalable cell therapy.
Mechanism of Action
Dopamine producing cells are implanted into the striatum to replace the lost dopaminergic neurons in these patients.
Unmet Medical Need
The current standard of care is L-DOPA, an oral medication that reduces the symptoms caused by Parkinson’s Disease. For many patients, this therapy loses its effectiveness over time and the symptoms of Parkinson’s increase in severity.
Project Objective
Pre-IND meeting and ready for GMP manufacturing
Major Proposed Activities
Human pluripotent stem cell expanded and differentiated dopaminergic precursor cells to treat Parkinson’s Disease at high quantity and high quality
Area of Impact
This candidate uses a 3D cell culture technology that addresses the manufacturing bottleneck and creates a high-quality scalable cell therapy.
Mechanism of Action
Dopamine producing cells are implanted into the striatum to replace the lost dopaminergic neurons in these patients.
Unmet Medical Need
The current standard of care is L-DOPA, an oral medication that reduces the symptoms caused by Parkinson’s Disease. For many patients, this therapy loses its effectiveness over time and the symptoms of Parkinson’s increase in severity.
Project Objective
Pre-IND meeting and ready for GMP manufacturing
Major Proposed Activities
- Produce material for 3D cell culture as well as cell banks at scale to support non-clinical animal studies and future clinical translation
- Produce dopaminergic neuron precursor cells in a 3D system for animal studies, compatible with the scale needed to meet patients demand
- Conduct non-clinical animal studies to define dose ranges and safety profile of the dopaminergic cells produced in the scalable 3D platform
Statement of Benefit to California:
The proposed program will develop a scalable manufacturing process to generate high quality dopamine producing cells with high survival and innervation capabilities post-transplantation, for the future implantation of these cells into patients suffering from PD. The ability to scale the process will lower the cost of manufacturing compared to standard methodologies leading to greater product accessibility to meet the large demand in California for these life-changing therapies.