Preclinical study of cell therapy for macular degeneration.

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AMD is a degenerative retinal disease that represents the leading cause of non-hereditary blindness in people over 65 years of age in developed countries.

The project
Who is behind

What is our goal?

Age-related macular degeneration (AMD) is an ophthalmological disease that causes progressive loss of visual acuity and blindness due to deterioration of the macula. The macula is a yellowish area of light-sensitive tissue at the back of the eye, in the center of the retina. This area provides visual acuity and allows us to perceive fine and small details. When the macula does not work properly, the areas in the center of the visual field begin to lose sharpness. Patients with advanced AMD may be affected in their ability to read, recognize faces, drive, etc., limiting many daily-life activities.

More than 800,000 people in Spain are currently affected of AMD, being this disease the main cause of blindness in adults in the developed countries. Smoking, advanced age, a family history of macular degeneration, a high level of blood cholesterol, among others, are factors predisposing to AMD. The prevalence of this degenerative disease associated to age is expected to grow in our increasingly long-lived societies. Despite its high prevalence, there are no effective treatments for the non-exudative form of the disease (dry AMD). For all this, it is essential to promote the development of new therapies that mitigate the high social and economic impact of AMD.

During the last decade, cell therapy has emerged as a new therapeutic alternative for degenerative diseases that cause blindness. Cell therapy consists in the replacement of damaged cells in the retina with new cells. The cells to be replaced can be photoreceptors or retinal pigment epithelium (RPE).

AMD begins with the degeneration and loss of RPE cells in the macula. For this reason, the replacement of dead cells by a new RPE has been proposed as a promising strategy for AMD treatment. Initially, an attempt was made to obtain RPE for transplantation from various sources: from a healthy area of the patient's own RPE, RPE from human fetuses, and RPE-derived cell lines; all with little success. More recently, very promising trials obtain RPE by differentiation in the laboratory from pluripotent cells (embryonic stem cells-ESC or induced pluripotent cells-iPS).

The latest clinical trials have been conducted in the United Kingdom, the United States, and Japan to evaluate the safety and efficacy of ESC- or iPS-derived RPE transplants. Their results have shown that transplantation is safe for the AMD patient, but the efficacy in terms of improving visual acuity is variable, and in some cases very minor improvement was achieved. There are numerous factors that must be adjusted to make RPE transplantation a regular treatment for the AMD patients who need it.

The main objective of this project is to produce a macular bio-patch of clinical quality for cell transplantation in patients with macular degeneration and to test its quality and utility for transplantation in a preclinical trial. Our aim is to obtain information on the safety and efficacy of the transplant, which will allow us to promote a future clinical trial that would ultimately be carried out in people affected by macular degeneration where, among other variables, the recovery of the visual acuity would be evaluated.

The specific objectives of this project are:

i)  Generate reprogrammed induced-pluripotent cell lines (iPS) from monocytes of healthy controls and characterize their human leukocyte antigens (HLA) profile.

ii)  Produce, under GMP conditions, a biocompatible graft of cells formed of the retinal pigment epithelium (RPE) derived from iPS cultured on a nanostructured fibrin-agarose hydrogel (NFAH)

iii)  Optimize the surgical technique and the surgical devices necessary to facilitate subretinal implant transplantation.

iv)  Evaluate the safety and efficacy of the iPS-derived RPE graft in experimental AMD models.

Abaout Precipita
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C/Pintor Murillo, 15
28100 Alcobendas (Madrid) - España
T.+(00 34) 91 425 09 09
info@precipita.es
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