Peptidomimetic therapy to promote central Nervous System repair

Descripción del proyecto

Terapia regenerativa pionera para el glaucoma

El glaucoma es una enfermedad neurodegenerativa progresiva. Se caracteriza por el daño del nervio óptico y la muerte de las células ganglionares de la retina, que transmiten la información visual del ojo al cerebro. Constituye la segunda causa de ceguera en el mundo, y las terapias actuales se centran sobre todo en reducir la presión intraocular, pero no logran restaurar la visión perdida. En el proyecto POPEYE, financiado por el Consejo Europeo de Investigación, se concebirá una terapia regenerativa pionera que favorecerá la recuperación funcional. La terapia emplea péptidos sintéticos que estimulan la regeneración axonal y la reconstrucción de los circuitos neuronales. Representa el primer intento de reparar y reconfigurar el nervio óptico en el glaucoma con alta precisión, una vía que ofrece esperanza a millones de pacientes en todo el mundo.

Objetivo

Vision enables 70% of our sensorial interactions with the outside world, making blindness a devastating condition with profound impact on quality of life. Glaucoma, the second leading cause of blindness globally after cataracts, affects over 80 million people worldwide, including more than 1 million in France alone. The disease impacts approximately 2% of the population over 40 years old and 10% of those over 70. With an aging global population, these numbers are projected to rise significantly in the coming years.
Glaucoma is fundamentally a neurological disorder characterized by optic nerve degeneration and death of Retinal Ganglion Cells (RGC), the crucial neurons that transmit visual information from the eye to the brain. The primary treatment approach focuses on lowering intraocular pressure (IOP) through medications like prostaglandins and beta-adrenergic receptor antagonists, or through surgical intervention. However, these treatments are purely preventative - they can only protect remaining healthy neurons and slow disease progression. They cannot reverse existing damage to the optic nerve or prevent RGC death in advanced cases. Moreover, not all forms of glaucoma are associated with elevated IOP, leaving some patients without effective treatment options. POPEYE introduces a groundbreaking approach to treating glaucoma that represents a significant departure from existing therapeutic strategies. Our innovation lies in developing a first-in-class therapeutic that directly promotes functional recovery through axon regeneration and proper neural circuit reconstruction.
The cornerstone of our innovation is the novel use of synthetic peptides to promote both axon regeneration and precise pathfinding. This dual action mechanism distinguishes our approach from all existing regenerative approaches, which have struggled with axon targeting accuracy.