Grant Award Details

Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models
Grant Number: 
TRAN1-10995
Project Objective: 
  • To investigate morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models and complete a pre-IND meeting with the FDA

Investigator: 
Type: 
PI
Disease Focus: 
Retinitis Pigmentosa
Vision Loss
Human Stem Cell Use: 
Embryonic Stem Cell
Cell Line Generation: 
Embryonic Stem Cell
Award Value: 
$4,769,039
Status: 
Active

Grant Application Details

Application Title: 
  • Morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models
Public Abstract: 

Translational Candidate

Retina organoid sheets (ROs) derived from CSC14 human embryonic stem cells (NIH registry line #0284) manufactured under GMP conditions

Area of Impact

Retinitis PIgmentosa (RP) (irreversible loss of photoreceptors) due to mutation of photoreceptors and/or other retinal genes

Mechanism of Action

Proposed mechanism of action is cell replacement, combined with trophic effects. Transplanted hESC-derived retina organoid sheets will mature into photoreceptors and integrate with the degenerate recipient’s retina. Such transplants have improved visual acuity and responses to flashes of light in the midbrain (superior colliculus) of immunodeficient retinal degenerate rats (two different models).

Unmet Medical Need

There is currently no treatment for retinitis pigmentosa which is designated an Orphan disease by the FDA. Therapies in current clinical trials only target trophic effects which are only effective in early stages to delay degeneration.

Project Objective

Pre-IND meeting

Major Proposed Activities

  • Establishment of Working Cell Bank, GMP implementation of retina organoid (RO) production, establish product specification and release criteria
  • Identify and demonstrate markers correlated with function after maturation in vitro; functional in vitro imaging (FLIM and HSpec)
  • In vivo pharmacology: demonstrate efficacy in immunodeficient and -competent rat model and in immunocompetent rabbit model of RP,
Statement of Benefit to California: 

Retinal diseases reduce the quality of life of patients, at significant cost to the health care system. The proposed replacement therapy is the only one that targets more mature disease stages of RP, for which no other therapy exists. An effective treatment will keep afflicted individuals productive, enhance State tax revenues and defray the healthcare cost burden to taxpayers. It will also lead to robust industry developments, effectively leading to job creation and tax benefits.