Clinical Translation of Autologous Regenerative Pluripotent Stem Cell Therapy for Blindness

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Grant Award Details

Grant Number:
Human Stem Cell Use:
Cell Line Generation:
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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.


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.