Stem Cell Therapy

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Stem cells are notable cells that are produced in the body and have the amazing potential to transform into many different cell types during growth and early life.

There are two main different types of stem cells that are most likely to be used for stem cell therapy:

  • Embryonic
  • Induced pluripotent (iPS cells). These cells are engineered in the lab.

Stem Cell Clinical Trials for Stargardt’s Disease

Ocata Therapeutics (formerly Advanced Cell Technology, ACT) have conducted a clinical trial in patients with advanced Stargardt's disease using human embryonic stem cell derived retinal pigment epithelial (RPE) cells. These cells were injected under the retina. RPE cells whose role is to support and nourish the photoreceptors (rods and cones) in the retina, degenerate in Stargardt's disease, often before photoreceptor cell loss. The idea is that by replacing healthy RPE cells, this will stop or slow down photoreceptor cell loss and thereby slow / halt vision loss.

Early results have been promising with a successful safety trial, conducted in the USA and UK.

Ocata Therapeutics has since been taken over by Astellas Pharma – with a new Phase I/II trial in Stargardt's disease of an improved RPE cell line being developed.

Opsis Therapeutics are developing a clinical trial where a patch consisting of retinal cells derived from stem cells will be placed at the back of the eye with the hope to restore vision. Dr. David Gamm, MD, PhD at the University of Wisconsin-Madison is leading this study with a team of researchers.

BlueRock Therapeutics ( are also developing retinal stem cell therapies for a range of inherited retinal diseases, including potentially Stargardt disease.