Transplantable 3-D retinas
Research with stem cell-derived constructs making strides as novel method to restore visual acuity
The development of a three-dimensional (3-D) transplantable retina may represent the greatest challenge that has ever been addressed in the area of stem cell-based tissue engineering. A team of researchers at AIVITA Biomedical led by Hans S. Keirstead, PhD, has made steady progress along the path and sees the launch of a clinical trial on the horizon.
Using human embryonic stem cells (hESCs), the group has successfully developed a complete retinal organoid consisting of laminated retinal progenitor cells and retinal pigment epithelium (RPE) and shown in preclinical studies that the injected material forms synaptic connections with the host and restores vision.
In 2016, AIVITA Biomedical received grants from both the National Eye Institute of the National Institutes of Health and the California Institute for Regenerative Medicine to develop the 3-D-transplantable retinas in collaboration with researchers at the Sue and Bill Gross Stem Cell Research Center at the University of California, Irvine.
The company is now gearing up the manufacturing operation to ensure it is clinically and commercially compliant.
Dr. Keirstead said he anticipates that entry into the clinical research phase may be as early as 2 years away.
“The cause for hope for transplanting a 3-D retina has never been greater,” explained Dr. Keirstead, chief executive officer, AIVITA Biomedical, Irvine, CA. “We have been on a relatively long journey, but are now at a point where we will be walking along a well-articulated path that will lead us to the beginning of our first in-human study.”
The target population for the 3-D retina is patients with degenerative diseases affecting the outer retina, such as age-related macular degeneration and retinitis pigmentosa.
The goal is to replace the diseased and non-functional photoreceptors and RPE with new cells that can establish functional connections with the inner neural retina in order to restore vision.