NeuBright, a purified allogeneic cell therapy product for treatment of Dry Age-related Macular Degeneration
Grant Award Details
Grant Type:
Grant Number:
TRAN1-11300
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$4,235,758
Status:
Active
Grant Application Details
Application Title:
NeuBright, a purified allogeneic cell therapy product for treatment of Dry Age-related Macular Degeneration
Public Abstract:
Translational Candidate
NeuBright is an allogeneic cryopreserved neural stem cell therapy product
Area of Impact
Dry Age-Related Macular Degeneration (AMD)
Mechanism of Action
Similar to RPE cells, NeuBright cells restore phagocytic function to the retina, secrete anti-inflammatory and trophic factors (VEGF and BDNF), maintain retinal integrity and prevent vision decline. Cells migrate radially from transplant site, self-renew to produce more cells and stimulate proliferation of endogenous host RPE cells. These properties allow the implanted NeuBright cells to exert protective effects over larger target areas and could potentially restore areas of geographic atrophy.
Unmet Medical Need
AMD is the leading cause of irreversible vision loss in people over 60 in the US with no effective therapie. AMD incidence israpidly increasing as the average age of the population increases. NeuBright cells will halt the process (slow progress, revision, etc) of vision loss.
Project Objective
Successful Pre-IND meeting
Major Proposed Activities
NeuBright is an allogeneic cryopreserved neural stem cell therapy product
Area of Impact
Dry Age-Related Macular Degeneration (AMD)
Mechanism of Action
Similar to RPE cells, NeuBright cells restore phagocytic function to the retina, secrete anti-inflammatory and trophic factors (VEGF and BDNF), maintain retinal integrity and prevent vision decline. Cells migrate radially from transplant site, self-renew to produce more cells and stimulate proliferation of endogenous host RPE cells. These properties allow the implanted NeuBright cells to exert protective effects over larger target areas and could potentially restore areas of geographic atrophy.
Unmet Medical Need
AMD is the leading cause of irreversible vision loss in people over 60 in the US with no effective therapie. AMD incidence israpidly increasing as the average age of the population increases. NeuBright cells will halt the process (slow progress, revision, etc) of vision loss.
Project Objective
Successful Pre-IND meeting
Major Proposed Activities
- Manufacturing process changes for more consistent and better procedural delivery of the NeuBright product.
- Animal study to confirm dose, delivery instrument and local immunosuppression regimen achieving >50% of eyes with product.
- Alignment with FDA on successful path to Phase 2 IND for geographic atrophy in AMD.
Statement of Benefit to California:
Dry age-related macular degeneration (AMD) is the most prevalent retinal degenerative disorder and represents a significant unmet medical with no therapy to slow progression or cure this debilitating disease. In California, a 2014 analysis estimated the cost due to vision loss and blindness at $7,592M and $8,054M respectively. NeuBright cells have preliminary evidence of efficacy and proposed changes should enhance patient access, commercial viability and accelerate path to market.