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Advancing a scalable and multifactorial exosome therapy to address the unmet needs of drug-induced hearing loss

Objetivo

Chemotherapy with platinum-based agents such as cisplatin has dramatically improved cancer survival, but at the cost of a severe side effect: profound hearing loss. Each year, millions of patients receive these drugs, and about half experience permanent auditory damage. This ototoxicity arises from multifactorial mechanisms involving oxidative stress, mitochondrial dysfunction, DNA damage, and inflammatory and apoptotic pathways, which together lead to irreversible hearing loss. The current standard of care remains purely compensatory, limited to hearing aids or cochlear implants, and no approved preventive therapy exists for adults or metastatic cancer patients.
Exogenus’ pioneering therapy, EXO-OTO-01, is a first-in-class exosome product derived from umbilical cord blood, a uniquely young and immunologically balanced source currently being explored for otoprotection. This origin confers distinct biological advantages, endowing EXO-OTO-01 with antioxidative, immunomodulatory, and potential regenerative properties that directly counteract the multifactorial damage mechanisms underlying hearing loss. The product is manufactured through a patented and GMP-compatible process, and preclinical models have already demonstrated significant otoprotection after local delivery.
This EIC Pre-Accelerator will enable us to de-risk EXO-OTO-01 by advancing both its technological maturity and investment readiness, paving the way for first-in-human studies post-project. The project will deliver GMP-like manufacturing, an optimized delivery route for clinical translation, and efficacy and local safety data, advancing EXO-OTO-01 from TRL 4 to early TRL 6. It will also strengthen regulatory, IP, and business readiness, including through market and business modelling.
Given the large unmet need and growing pharmaceutical interest in hearing loss prevention, EXO-OTO-01 represents a transformative opportunity in otoprotection, positioning Europe at the forefront of exosome therapeutics.

Ámbito científico (EuroSciVoc)

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Programa(s)

Tema(s)

Régimen de financiación

HORIZON-CSA - HORIZON Coordination and Support Actions

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Convocatoria de propuestas

(se abrirá en una nueva ventana) HORIZON-WIDERA-2025-02

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Coordinador

EXOGENUS THERAPEUTICS SA
Aportación neta de la UEn
€ 500 000,00
Dirección
PARQUE BIOCANT NUCLEO 04 LOTE 2
3060-197 CANTANHEDE
Portugal
Pyme
Sí
Tipo de actividad
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Enlaces
Coste total
Sin datos