Retinitis pigmentosa (RP) is a rare genetic disease of the eye characterized clinically by the loss of rod photoreceptor cells, followed by cone-cell degeneration. As rod cell function degenerates, impaired night vision typically appears as an initial symptom. When rod photoreceptors die, cone photoreceptors are destabilized, resulting in progressive loss of visual acuity and color/central vision. The principal collaborators developed a cell-based therapeutic approach to stop retinal cell degeneration. Work in animal models has demonstrated the ability of grafted retinal-derived, lineage-restricted progenitor cells (RPCs) to promote survival and function of dystrophic host photoreceptors. The lead investigators and others have demonstrated that injected donor RPCs survive in the vitreous and migrate to the retina, where they integrate with the host cellular architecture. As lineage-restricted progenitor cells, these engrafted RPCs are able to differentiate further into rod photoreceptors, providing additional support to the dependent cone cells. The therapeutic hypothesis is that the release of trophic factors and engraftment by donor RPCs should rescue host cone cells, improving existing vision and delaying or preventing total blindness. In a pre-Investigational New Drug (IND) Application meeting, the U.S. Food and Drug Administration (FDA) identified additional studies needed to support submission of a full IND data package. Studies were performed by TRND and the collaborator to support submission of a full IND Application package. TRND researchers completed a key biodistribution study of the formulated retinal cells (jCell) in animals. The results of these studies were incorporated into an IND Application filing by the collaborators start-up company, jCyte, Inc. The IND was cleared by the FDA for use in human clinical trials. jCyte, Inc., now will be able to continue development of jCell using internal resources.

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