Regenerative medicine biotech Endogena Therapeutics has received the green light from the US FDA for the Investigational New Drug application for its compound, EA-2351, paving the way for first-in-human studies in 2024. The compound is thought to hold promise for patients suffering from geographic atrophy, an advanced form of age-related macular degeneration (AMD).

Endogena is focused on discovering and developing endogenous regenerative medicines for tissue and organ repair. The company’s approach involves the selective regulation of endogenous adult stem and progenitor cells using small molecules, as it seeks to treat degenerative conditions related to aging and genetic disorders.

The EA-2351 program now follows Endogena’s EA-2353 program for retinitis pigmentosa into clinical trials. Both programs target specific cell populations through different pathways, relying on their unique chemical structures. While EA-2353 focuses on activating specific cells related to retinitis pigmentosa, EA-2351 targets retinal pigment epithelial cells, which are essential for regenerating and restoring photoreceptor function.

AMD is a leading cause of irreversible vision loss in the developed world, impacting nearly 9% of individuals over the age of 45. The “dry” form of AMD, characterized by atrophic cell death in the central retina or macula, accounts for 80-90% of the total AMD population.

“We look forward to continuing to develop yet another potential therapy for a condition with a high unmet medical need, thereby providing hope for patients affected by AMD,” said Dr Matthias Steger, PhD, CEO of Endogena. “Many of us will know someone touched by this terrible disease, which makes our work even more pertinent. Our resilience, clear purpose, and the financial support from our shareholders have enabled us to reach this point, and will help pave our way to future success.”