Peptidomimetic therapy to promote central Nervous System repair

Projektbeschreibung

Wegweisende regenerative Glaukomtherapie

Das Glaukom ist eine fortschreitende neurodegenerative Erkrankung, die durch eine Schädigung des Sehnervs und das Absterben der retinalen Ganglienzellen, welche die visuellen Informationen vom Auge zum Gehirn weiterleiten, gekennzeichnet. Es handelt sich um die weltweit zweithäufigste Ursache für Erblindung. Die derzeitigen Therapien zielen hauptsächlich auf die Senkung des Augeninnendrucks ab, können aber das verlorene Sehvermögen nicht wiederherstellen. Das ERC-finanzierte Projekt POPEYE wird eine wegweisende regenerative Therapie entwickeln, welche die funktionelle Wiederherstellung fördern soll. Bei der Therapie werden synthetische Peptide eingesetzt, um die Axonregeneration und den Wiederaufbau neuronaler Schaltkreise anzuregen. Dies stellt den ersten Versuch dar, bei einem vorhandenen Glaukom den Sehnerv mit hoher Präzision zu reparieren und neu zu vernetzen, so dass Millionen von Patientinnen und Patienten weltweit Hoffnung schöpfen können.

Ziel

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.