Optogenetic Therapy for Treatment of Geographic Atrophy
Grant Award Details
Grant Type:
Grant Number:
TRAN1-15341
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$3,998,930
Status:
Active
Grant Application Details
Application Title:
Optogenetic Therapy for Treatment of Geographic Atrophy
Public Abstract:
Translational Candidate
Optogenetic gene therapy for patients with geographic atrophy age related macular degeneration.
Area of Impact
Blindness from geographic atrophy age related macular degeneration
Mechanism of Action
Gene therapy to deliver optogenetic protein to the targeted cells of the retina to restore vision.
Unmet Medical Need
Geographic atrophy (GA) age related macular degeneration is an advanced form of Age-related Macular Degeneration (AMD), is a very common disease of the eye retina affecting 1-1.5 million patients in the United States (US). Prevalence is expected to double by 2040.
Project Objective
Pre-IND completion.
Major Proposed Activities
Optogenetic gene therapy for patients with geographic atrophy age related macular degeneration.
Area of Impact
Blindness from geographic atrophy age related macular degeneration
Mechanism of Action
Gene therapy to deliver optogenetic protein to the targeted cells of the retina to restore vision.
Unmet Medical Need
Geographic atrophy (GA) age related macular degeneration is an advanced form of Age-related Macular Degeneration (AMD), is a very common disease of the eye retina affecting 1-1.5 million patients in the United States (US). Prevalence is expected to double by 2040.
Project Objective
Pre-IND completion.
Major Proposed Activities
- Confirmatory studies in visual response after treatment with optogenetic protein.
- Confirmed appropriate cell transduction, expression, and preliminary safety.
- Process Developoment and manufacturing of product (CMC).
Statement of Benefit to California:
Age-related macular degeneration is a major cause of vision loss in older Americans. It is estimated that >100,000 Californians are blind from Geographic Atrophy (GA), one of the two advanced forms of the disease. Patients experience gradual loss of central vision resulting in loss of the ability to read, recognize facies drive and loss of independence. There are no treatments to reverse GA or restore lost vision. We are developing a potential breakthrough treatment that may restore lost vision.