SubsidieMeestersPannexin 1 – a novel target for pediatric orphan epilepsy
PTI5803 is a first-in-class drug that specifically blocks the activated Pannexin 1 channel, effectively reducing seizures in ultra-resistant pediatric epilepsy while preserving cognitive functions.
Projectdetails
Introduction
Epilepsy is a group of syndromes with high unmet medical need, especially in children as frequent seizures affect the immature brain and its development.
Drug Resistance
In some orphan syndromes, drug resistance is 70% with dramatic consequences for patients and their families.
Treatment Goals
Novel treatments must:
- Prevent the negative effects of seizures
- Minimize off-target effects on brain development and cognitive functions
Proposed Solution
Our solution is the specific blockade of the activated form of the Pannexin 1 (Panx1) channel using a reformulated form of probenecid: PTI5803.
Compound Efficacy
This compound, a first-in-class drug, inhibits seizures preclinically in over 80% of cases without hampering cognitive functions.
Adaptation for Children
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.
Market Impact
PTI5803 has the potential to disrupt the market of paediatric epilepsy.
Financiële details & Tijdlijn
Financiële details
| Subsidiebedrag | € 2.499.500 |
| Totale projectbegroting | € 4.078.906 |
Tijdlijn
| Startdatum | 1-10-2023 |
| Einddatum | 31-3-2026 |
| Subsidiejaar | 2023 |
Partners & Locaties
Projectpartners
- PANNTHERAPIpenvoerder
Land(en)
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Vergelijkbare projecten uit andere regelingen
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|---|---|---|---|---|
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Targeting epilepsy with phototherapeuticsThis project aims to develop targeted photoactivatable drugs to control focal epilepsy, minimizing side effects and potentially transforming treatment options for intractable cases. | ERC Starting... | € 1.499.375 | 2022 | Details |
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Versatile Polypeptide-based Intranasal Drug Delivery Platform(s) to Tackle CNS Disorders.POLYBRAINT aims to develop an innovative intranasal drug delivery platform to effectively transport biological agents across the blood-brain barrier for treating CNS disorders. | ERC Proof of... | € 150.000 | 2022 | Details |
Targeting Epilepsy Ictogenesis Process Through Pannexin-1 Modulation
The project aims to develop a new generation of antiepileptic drugs targeting a novel mechanism to improve treatment for pharmacoresistant epilepsy patients.
Targeting epilepsy with phototherapeutics
This project aims to develop targeted photoactivatable drugs to control focal epilepsy, minimizing side effects and potentially transforming treatment options for intractable cases.
Senolytics repurposing in childhood refractory epilepsies
EpiSen aims to develop a precision pharmacological strategy to selectively eliminate mutated cells in Focal Cortical Dysplasia, potentially reducing drug-resistant seizures in children.
NOVEL RESUCE-MEDICATION BASED SYSTEM TO PREVENT EPILEPTIC SEIZURES
Het project onderzoekt de haalbaarheid van een innovatief apparaat dat epileptische aanvallen voorspelt en voorkomt, ter verbetering van de levenskwaliteit.
Versatile Polypeptide-based Intranasal Drug Delivery Platform(s) to Tackle CNS Disorders.
POLYBRAINT aims to develop an innovative intranasal drug delivery platform to effectively transport biological agents across the blood-brain barrier for treating CNS disorders.