CORDIS - Forschungsergebnisse der EU
CORDIS

Pannexin 1 – a novel target for pediatric orphan epilepsy

Ziel

Epilepsy is a group of syndromes with high unmet medical need, especially in children as frequent seizures affect the immature brain and its development. In some orphan syndromes, drug resistance is 70% with dramatic consequences for patients and their families. Novel treatments must prevent the negative effects of seizures, but also minimize off-target effects on brain development and cognitive functions. Our solution is the specific blockade of the activated form of the Pannexin 1 (Panx1) channel using a reformulated form of probenecid: PTI5803. This compound, a first-in-class drug, inhibits seizures preclinically in >80% of cases without hampering cognitive functions. Designed to be adapted to children (from 6 months of age) and to improve the bioavailability, PTI5803 is a highly specific breakthrough product to counteract the pathological activation of Panx1 in patients with an ultra-resistant orphan epilepsy, with the potential to disrupt the market of paediatric epilepsy.

Schlüsselbegriffe

Koordinator

PANNTHERAPI
Netto-EU-Beitrag
€ 2 499 500,00
Adresse
10 RUE DESCARTES
30000 NIMES
Frankreich

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KMU

Die Organisation definierte sich zum Zeitpunkt der Unterzeichnung der Finanzhilfevereinbarung selbst als KMU (Kleine und mittlere Unternehmen).

Ja
Region
Occitanie Languedoc-Roussillon Gard
Aktivitätstyp
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Links
Gesamtkosten
€ 4 078 906,00