Clinical Translation of Autologous Regenerative Pluripotent Stem Cell Therapy for Blindness
Grant Award Details
Grant Type:
Grant Number:
CLIN1-14602
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:
$6,000,000
Status:
Active
Grant Application Details
Application Title:
Clinical Translation of Autologous Regenerative Pluripotent Stem Cell Therapy for Blindness
Public Abstract:
Therapeutic Candidate or Device
The therapeutic candidate is a patient specific (autologous) induced pluripotent stem cell derived retinal pigment epithelium (AiPSC-RPE) product.
Indication
AiPSC-RPE cell product will be indicated for the treatment of maculopathies related to RPE atrophy.
Therapeutic Mechanism
The proposed therapy is an autlogous cell product and intended to be used as a cell replacement to the endogenous RPE layer in patients with advanced RPE atrophy. The AiPSC-RPE cells are suspended at a concentration of 250,000, 500,000, or 1,000,000 cells per 150 uL of a balanced salt solution enriched with bicarbonate, dextrose and glutathione, administered via transvitreal sub-macular transplantation.
Unmet Medical Need
Several degenerative diseases of the eye cause permanent vision loss due to RPE cell dysfunction. There are no available treatments for these diseases. The proposed AiPSC-RPE cell product may rescue vision, providing the potential to address one of the world's major unmet medical deeds.
Project Objective
Completed IND submission to the FDA
Major Proposed Activities
The therapeutic candidate is a patient specific (autologous) induced pluripotent stem cell derived retinal pigment epithelium (AiPSC-RPE) product.
Indication
AiPSC-RPE cell product will be indicated for the treatment of maculopathies related to RPE atrophy.
Therapeutic Mechanism
The proposed therapy is an autlogous cell product and intended to be used as a cell replacement to the endogenous RPE layer in patients with advanced RPE atrophy. The AiPSC-RPE cells are suspended at a concentration of 250,000, 500,000, or 1,000,000 cells per 150 uL of a balanced salt solution enriched with bicarbonate, dextrose and glutathione, administered via transvitreal sub-macular transplantation.
Unmet Medical Need
Several degenerative diseases of the eye cause permanent vision loss due to RPE cell dysfunction. There are no available treatments for these diseases. The proposed AiPSC-RPE cell product may rescue vision, providing the potential to address one of the world's major unmet medical deeds.
Project Objective
Completed IND submission to the FDA
Major Proposed Activities
- Manufacture 6 AiPSC-RPE cell products for potential use in a Phase I clinical trial. Conduct AiPSC-RPE final product manufacturing runs.
- Assess safety by conducting IND enabling non-clinical GLP safety/toxicology/biodistribution studies.
- Generation of materials and regulatory review of AiPSC-RPE cell products to support an IND submission to the FDA.
Statement of Benefit to California:
Vision impairment impacts both the length and quality of life. By providing a cell therapy that may rescue vision with minimally invasive surgery and does not require immune suppression, the proposed AiPSC-RPE product provides the potential to address one of the major unmet medical needs in California and the developed world at large.
