Pannexin 1 – a novel target for pediatric orphan epilepsybroad
PANNEXIN · Horizon Europe grant · 2023-10-01–2026-03-31
EC contribution
Total cost
Beneficiaries
About the data
Source: CORDIS (official EU open data), Horizon Europe. Framework HORIZON · call HORIZON-EIC-2023-ACCELERATOR-01 · scheme HORIZON-EIC-ACC-BF · topic HORIZON-EIC-2023-ACCELERATOROPEN-01. CORDIS record →
Objective
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.
Beneficiaries (1)
| Organisation | Country | Role | EC contribution | SME |
|---|---|---|---|---|
| PANNTHERAPI | FR | coordinator | €2,499,500 | Yes |
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