Extracellular Vesicle-Based Therapy for Corneal Scars
Grant Award Details
Grant Type:
Grant Number:
TRAN1-14649
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:
$5,779,276
Status:
Active
Grant Application Details
Application Title:
Extracellular Vesicle-Based Therapy for Corneal Scars
Public Abstract:
Translational Candidate
Extracellular vesicles derived from stem cells for the treatment of corneal scars.
Area of Impact
The candidate aims to restoring vision by reducing the need for corneal transplant and the associated blinding complications.
Mechanism of Action
The therapeutic candidate is an extracellular vesicle (EV)-based therapy that could regenerate normal cornea tissue and reduce corneal scars without the need of corneal transplant.The unique population of EVs have anti-inflammatory and anti-fibrotic activities in addition to their regenerative property. The effect of EVs are likely acted via their microRNA cargo.
Unmet Medical Need
Blinding complications such as infection, glaucoma and retinal detachment are associated with corneal transplant. By reducing corneal scars, the vision will improve without the need for a corneal transplant, which will also greatly reduce the impact of global severe shortage of corneal tissues.
Project Objective
Submit a pre-IND package
Major Proposed Activities
Extracellular vesicles derived from stem cells for the treatment of corneal scars.
Area of Impact
The candidate aims to restoring vision by reducing the need for corneal transplant and the associated blinding complications.
Mechanism of Action
The therapeutic candidate is an extracellular vesicle (EV)-based therapy that could regenerate normal cornea tissue and reduce corneal scars without the need of corneal transplant.The unique population of EVs have anti-inflammatory and anti-fibrotic activities in addition to their regenerative property. The effect of EVs are likely acted via their microRNA cargo.
Unmet Medical Need
Blinding complications such as infection, glaucoma and retinal detachment are associated with corneal transplant. By reducing corneal scars, the vision will improve without the need for a corneal transplant, which will also greatly reduce the impact of global severe shortage of corneal tissues.
Project Objective
Submit a pre-IND package
Major Proposed Activities
- Identification of biomarkers and functional units of therapeutic candidate which will serve as basis of potency assays.
- Optimization of route of delivery and dosing, pharmacokinetics and pilot safety.
- Establishment of GMP compliant master cell bank and scalable extracellular vesicle purification process.
Statement of Benefit to California:
California is the most populated state in the USA. The number of residents with corneal scars may disproportionately increase as a result of multiple environmental and occupational factors. A safe treatment to restore vision is an important benefit to the people of California. Our project will further benefit California through the training of new stem-cell researchers, create more jobs, and attract funding from the federal government and investment from the private sector.