Cellular Dynamics International, has announced that the National Eye Institute (NEI), a division of the National Institutes of Health (NIH), awarded the company a $1.2 million contract to manufacture clinically compatible induced pluripotent stem cells (iPSCs) and iPSC-derived human retinal pigment epithelial (RPE) cells. These cells will be manufactured from individuals suffering from dry age-related macular degeneration (AMD) and will be used for investigational new drug (IND)-enabling studies. Once the IND is approved, the same procedures will be used to generate clinical-grade iPSC-derived RPE tissue for transplantation into AMD patients. This process, known as autologous cellular therapy, would be the first of its kind in the U.S.
CDI will use its expertise to reprogram skin and blood samples from individuals with AMD to create clinically compatible, autologous iPSCs, which are genetically identical to the individual. As a genetic match to the patient, these cells are intended to reduce the risk of transplant rejection. NEI researchers plan to use these cells as part of their pre-clinical process to develop the first autologous cell transplantation treatment for dry AMD.
- The clinically compatible RPE cells resulting from CDI’s protocol development and optimization, when manufactured under cGMP conditions, would likely be the first iPSC-based, autologous cellular therapeutic candidates transplanted into humans in a Phase I clinical trial in the U.S.
- The contract funds the manufacture of clinically compatible iPSCs and the development and manufacture of clinically compatible iPSC-derived human RPE cells by CDI. The RPE manufacturing process is based on iPSC-RPE differentiation methods developed and authenticated by NEI.
- CDI will use existing NEI differentiation protocols to develop and optimize the methods for manufacturing RPE cells and provide NEI with cGMP-ready protocols and training for their manufacture.
- Researchers at NEI will use the RPE cells that CDI manufactures for preclinical studies in preparation for a clinical trial that will test these cells in patients with dry AMD. AMD is the leading cause of vision loss in people aged 60 and older, and as many as 11 million Americans have some form of macular degeneration, according to the Bright Focus Foundation.
- There are currently no FDA-approved therapies for dry AMD, which accounts for about 90% of AMD cases. Therapies for wet AMD require repeated eye injections that slow or, at best, arrest disease progression. Revenues for wet AMD products were more than $5.75 billion in 2013.
Bob Palay, chief executive officer of CDI, said, “AMD is a debilitating disease. It begins with vision loss in the center of the visual field and often progresses to nearly total vision loss. CDI is delighted to be selected by the NEI to develop autologous RPEs for treatment of AMD. Our goal is to provide Dr. Bharti and his fellow medical researchers with transplantable manufactured RPEs for the treatment of this major cause of blindness. We are excited to enable these researchers’ efforts to help patients regain their vision.”
Chris Parker, chief commercial officer of CDI said, “CDI has a unique ability to consistently manufacture large numbers of iPSCs and human cells to very tight specifications, including under cGMP conditions. CDI’s reprogramming platform enables us to develop autologous iPSC-based therapies, which would be a first in the U.S. We look forward to working with the NEI to successfully complete this work and progress through Phase I clinical development.”
Sheldon Miller, Ph.D., scientific director at the NEI, said, “This is a first step towards our phase I IND enabling studies. There are a small number of facilities in the U.S. with the capacity to generate iPSCs and differentiate them into multiple lineages. We are eager to begin testing clinically compatible patient-derived RPE cells in laboratory models of retinal degeneration.”
Kapil Bharti, Ph.D., Earl Stadtman investigator and lead scientist on this project at NEI, said, “All of us at NEI, including the basic research, preclinical, and clinical teams, are committed to the success of this IND. We are grateful to the NIH Common Fund and the NEI Intramural Program for their support.”