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hOTX2 is a biologic therapy in development by BrainEver for the treatment of glaucoma. The therapy is based on the OTX2 (Orthodenticle Homeobox 2) homeoprotein, a transcription factor that serves as a critical maintenance and survival factor for retinal ganglion cells (RGCs). In glaucoma, these neurons degenerate, leading to irreversible vision loss. The mechanism of action involves the uptake of exogenous OTX2 protein by retinal ganglion cells, where it promotes cell survival and protects against neurodegeneration. Preclinical proof-of-concept studies have demonstrated that OTX2 can protect RGCs from damage and preserve visual acuity. The program is being developed through a partnership between BrainEver SAS, the Collège de France, CNRS, and Inserm, exploring both protein and gene therapy delivery strategies.
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