Gene-corrected human microglia for the treatment of adult onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)
Grant Award Details
Grant Type:
Grant Number:
DISC2-15120
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$1,417,948
Status:
Active
Grant Application Details
Application Title:
Gene-corrected human microglia for the treatment of adult onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)
Public Abstract:
Research Objective
The research proposed here will support the development of NGL-101, a cell therapy for treating adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
Impact
Successful development of NGL-101 will result in a life-altering therapy for patients suffering from ALSP and provide proof of concept for treating other microglia diseases.
Major Proposed Activities
The research proposed here will support the development of NGL-101, a cell therapy for treating adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
Impact
Successful development of NGL-101 will result in a life-altering therapy for patients suffering from ALSP and provide proof of concept for treating other microglia diseases.
Major Proposed Activities
- Demonstrate the in vitro safety and efficacy of gene corrected iPSC-derived microglia.
- Demonstrate preclinical efficacy and safety of gene corrected iPSC-derived microglia.
Statement of Benefit to California:
Novoglia, Inc. is a California-based company, as are the two primary contractors, ClearPoint Neuro and Translucence Biosystems. Funding of this work will provide direct economic benefit to these companies and their employees in addition to providing needed therapies to California citizens suffering from microglia diseases.