Name:
Institution:
Type:
PI
Grant Award Details
Optogenetic therapy for treating retinitis pigmentosa and other inherited retinal diseases
Grant Type:
Grant Number:
TRAN1-13342
Project Objective:
To conduct a well-prepared pre-IND meeting with FDA for an AAV-mediated gene therapy expressing an engineered channelrhodopsin for the treatment of retinitis pigmentosa.
Investigator:
Disease Focus:
Retinitis Pigmentosa
Vision Loss
Human Stem Cell Use:
Somatic Cell
Award Value:
$3,999,553
Status:
Active
Grant Application Details
Application Title:
- Optogenetic therapy for treating retinitis pigmentosa and other inherited retinal diseases
Public Abstract:
Translational Candidate
Ray-001 is an AAV gene therapy delivering a light sensitive gene to treat patients with advanced Retinitis Pigmentosa.
Area of Impact
Retinitis pigmentosa (RP) is a genetic disease that causes retinal degeneration leading to near or complete blindness for most patients.
Mechanism of Action
Ray Therapeutics' therapy (Ray-001) delivers a potent transgene with high sensitivity to light incoming to the eye, and high dynamic range. Ray-001 has been shown to effectively treat the target retinal neurons. Light activation of the protein delivered by Ray-001 results in a signal being sent to the visual cortex of the brain.
Unmet Medical Need
Ray-001 will treat patients with advanced retinitis pigmentosa (RP) and other inherited retinal diseases (IRD) who currently have no other approved treatment.
Project Objective
pre-IND meeting
Major Proposed Activities
- Preclinical animal studies to further evaluate safety and efficacy
- Manufacturing process and analytical development
- Preparation for a pre-IND meeting
Statement of Benefit to California:
Retinitis pigmentosa (RP) is a progressively debilitating disease which leads to blindness. Of the approximate 10,000 patients living with RP in California, many have advanced disease, to the point of total loss of visual acuity. Most of these patients need to receive healthcare benefits, special living assistance, and suffer from loss of financial independence. Ray-001 represents a potential breakthrough treatment for a high unmet medical need for RP patients to improve their quality of life.
