DEVELOPMENT TOWARDS THERAPY OF MACULAR DEGENERATION OF RETINA BY DERIVATIVES OF HUMAN PLURIPOTENT STEM CELLS

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216224%3A14110%2F24%3A00136351" target="_blank" >RIV/00216224:14110/24:00136351 - isvavai.cz</a>

Výsledek na webu

—

DOI - Digital Object Identifier

—

Jazyk výsledku

angličtina

Název v původním jazyce

DEVELOPMENT TOWARDS THERAPY OF MACULAR DEGENERATION OF RETINA BY DERIVATIVES OF HUMAN PLURIPOTENT STEM CELLS

Popis výsledku v původním jazyce

Regenerative medicine in the field of ophthalmology was revolutionized in the 1990s through stem cell-based therapies focusing on treating corneal diseases1. However, there still exist numerous incurable diseases associated with the perceptive part itself, the retina. The pathogenesis of the multiple forms of retinopathies is often associated with the dysfunction and loss of retinal cell types, including retinal pigment epithelium (RPE). Our objective is, thus, to establish for the first time in the Czech Republic the current Good Manufacturing Practise (cGMP) compliant methodological and regulatory grounds for producing a key component of the Advanced Therapy Medicinal Product – human pluripotent stem cell-derived RPE cells2. By doing so, we aim to contribute to the advancement of regenerative medicine, particularly in the treatment of retinal diseases. In our ongoing research, we have determined the optimal conditions for differentiation of hPSC into RPE cells, utilizing established protocols as a foundational reference. We have started with the establishment of the morphological, molecular, and functional criteria to assess the efficacy of the differentiation process, alongside a comprehensive evaluation of the genetic stability to ensure their safety. Our next steps involve the crucial transfer of this methodology to cGMP conditions, laying the groundwork for the translation of our findings into the realm of clinical applications.

Název v anglickém jazyce

DEVELOPMENT TOWARDS THERAPY OF MACULAR DEGENERATION OF RETINA BY DERIVATIVES OF HUMAN PLURIPOTENT STEM CELLS

Popis výsledku anglicky

Regenerative medicine in the field of ophthalmology was revolutionized in the 1990s through stem cell-based therapies focusing on treating corneal diseases1. However, there still exist numerous incurable diseases associated with the perceptive part itself, the retina. The pathogenesis of the multiple forms of retinopathies is often associated with the dysfunction and loss of retinal cell types, including retinal pigment epithelium (RPE). Our objective is, thus, to establish for the first time in the Czech Republic the current Good Manufacturing Practise (cGMP) compliant methodological and regulatory grounds for producing a key component of the Advanced Therapy Medicinal Product – human pluripotent stem cell-derived RPE cells2. By doing so, we aim to contribute to the advancement of regenerative medicine, particularly in the treatment of retinal diseases. In our ongoing research, we have determined the optimal conditions for differentiation of hPSC into RPE cells, utilizing established protocols as a foundational reference. We have started with the establishment of the morphological, molecular, and functional criteria to assess the efficacy of the differentiation process, alongside a comprehensive evaluation of the genetic stability to ensure their safety. Our next steps involve the crucial transfer of this methodology to cGMP conditions, laying the groundwork for the translation of our findings into the realm of clinical applications.

Druh

O - Ostatní výsledky

CEP obor

—

OECD FORD obor

10605 - Developmental biology

Projekt

<a href="/cs/project/NU22-08-00629" target="_blank" >NU22-08-00629: Vývoj postupů pro léčbu makulární degenerace sítnice deriváty lidských pluripotentních kmenových buněk</a><br>

Návaznosti

P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)<br>S - Specificky vyzkum na vysokych skolach

Rok uplatnění

2024

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů