Name:
Institution:
Type:
PI
Grant Award Details
Allogeneic iPSC derived Dopaminergic Drug Product for Parkinson's disease
Grant Type:
Grant Number:
CLIN1-14300
Project Objective:
- The objectives of this award are to: 1) complete a NHP study to evaluate delivery of the RNDP-001 drug product (iPSC-derived dopaminergic precursor cells) into the putamen using an MR-compatible delivery device, 2) to develop a glial maturation assay and single cell RNAseq assay to further characterize the RNDP-001 product, 3) to prepare and submit an IND, and 4) to carry out start-up activities for a clinical trial in Parkinson's disease patients.
Investigator:
Disease Focus:
Neurological Disorders
Parkinson's Disease
Human Stem Cell Use:
iPS Cell
Award Value:
$4,000,000
Status:
Active
Grant Application Details
Application Title:
- Allogeneic iPSC derived Dopaminergic Drug Product for Parkinson's disease
Public Abstract:
Therapeutic Candidate or Device
Allogeneic iPSC derived dopamine progenitors delivered to the brain of Parkinson's disease patients
Indication
Idiopathic Parkinson's disease
Therapeutic Mechanism
The cellular product reconstitutes dopaminergic neuron circuits lost owing to the disease
Unmet Medical Need
There are currently disease modifying therapies. This approach is intended to be disease modifying.
Project Objective
To submit and have an IND cleared.
Major Proposed Activities
- FDA requested non-human primate study of cells delivered to brain with device
- FDA requested glial maturation assay development
- FDA recommended single cell RNA sequencing data
Statement of Benefit to California:
If successful the California based Ryne Bio's cellular drug product, RNDP-001, will be available to treat and modify disease of Parkinson's patients in California including in areas historically underserved. Ryne anticipates establishing its headquarters and hiring in California with the potential to have economic benefit through job creation.
