SubsidieMeestersLCTB-21: A DYRK1A protein kinase inhibitor for treatment of cognitive decline in Down Syndrome.
Perha Pharmaceuticals aims to outlicense LCTB-21, a DYRK1A inhibitor, to improve cognitive function and autonomy in children with Down Syndrome after successful phase 2a trials.
Projectdetails
Introduction
All people with Down Syndrome (DS) suffer from a certain level of impaired learning and memory (cognitive disorders) from childhood onwards, greatly impacting their autonomy and societal integration. This is becoming an increasingly urgent issue as people with DS are more often outliving their caretakers, yet an adequate treatment has not yet been developed.
Cognitive Decline and DYRK1A
Studies have revealed that the cognitive decline in people with DS is associated with overexpression of the DYRK1A protein kinase in the brain.
Therapeutic Development
Perha Pharmaceuticals has developed a novel therapeutic compound, Leucettinib-21 (LCTB-21), which acts as a DYRK1A inhibitor that can reduce cognitive decline in children with DS. Ultimately, this will increase the autonomy of people with DS, resulting in better societal integration.
Licensing Strategy
Perha Pharmaceuticals aims to outlicense LCTB-21 to a large pharmaceutical partner (e.g. Otsuka Pharmaceuticals, Intra-Cellular Therapies) following a successful phase 2a clinical trial.
Financiële details & Tijdlijn
Financiële details
| Subsidiebedrag | € 2.500.000 |
| Totale projectbegroting | € 11.359.709 |
Tijdlijn
| Startdatum | 1-6-2022 |
| Einddatum | 30-6-2026 |
| Subsidiejaar | 2022 |
Partners & Locaties
Projectpartners
- PERHA PHARMACEUTICALS SASpenvoerder
Land(en)
Vergelijkbare projecten binnen EIC Accelerator
| Project | Regeling | Bedrag | Jaar | Actie |
|---|---|---|---|---|
Dementia/Alzheimer Drug Attributed to Cystatin-C and other mutations – precision medicine drug developmentATI's AT-001 aims to stop and revert dementia by preventing protein aggregation, with promising Phase IIa results leading to upcoming registration trials and POC studies for commercialization. | EIC Accelerator | € 2.500.000 | 2023 | Details |
Developing a novel Parkinson's disease drug using small molecule mimetics of Glial cell line-Derived Neurotrophic Factor (GDNF)GeneCode is developing an oral anti-PD drug that mimics GDNF peptide pathways to restore dopamine neurons and alleviate symptoms in Parkinson's patients, with potential applications for other conditions. | EIC Accelerator | € 1.636.639 | 2024 | Details |
Dementia/Alzheimer Drug Attributed to Cystatin-C and other mutations – precision medicine drug development
ATI's AT-001 aims to stop and revert dementia by preventing protein aggregation, with promising Phase IIa results leading to upcoming registration trials and POC studies for commercialization.
Developing a novel Parkinson's disease drug using small molecule mimetics of Glial cell line-Derived Neurotrophic Factor (GDNF)
GeneCode is developing an oral anti-PD drug that mimics GDNF peptide pathways to restore dopamine neurons and alleviate symptoms in Parkinson's patients, with potential applications for other conditions.
Vergelijkbare projecten uit andere regelingen
| Project | Regeling | Bedrag | Jaar | Actie |
|---|---|---|---|---|
Senolytics repurposing in childhood refractory epilepsiesEpiSen aims to develop a precision pharmacological strategy to selectively eliminate mutated cells in Focal Cortical Dysplasia, potentially reducing drug-resistant seizures in children. | ERC Proof of... | € 150.000 | 2023 | Details |
Do T cells link loss and gain-of-function mechanism in C9orf72 ALS/FTD?This project investigates the role of T cells in the pathogenesis of ALS and FTD due to C9orf72 mutations, aiming to uncover mechanisms for new biomarkers and therapeutic targets. | ERC Starting... | € 1.498.610 | 2024 | Details |
Repurposing a human drug to treat Alzheimer’s diseaseThis project aims to validate an approved drug that inhibits tau accumulation in Alzheimer's, advancing it towards commercialization as a novel treatment for the disease. | ERC Proof of... | € 150.000 | 2022 | Details |
Unlocking cognition by Pulsatile GnRH in Alzheimer's DiseasEThe UPGRADE project aims to enhance cognitive performance in Alzheimer's patients by testing the safety and efficacy of pulsatile GnRH therapy based on promising preclinical results. | ERC Proof of... | € 150.000 | 2023 | Details |
Preclinical development of new nucleoside-based drug against leukemiaThe project aims to develop Carbacitabin (CAB) as a stable, effective treatment for high-risk leukemia patients through preclinical optimization, safety studies, and commercial preparation. | ERC Proof of... | € 150.000 | 2024 | Details |
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.
Do T cells link loss and gain-of-function mechanism in C9orf72 ALS/FTD?
This project investigates the role of T cells in the pathogenesis of ALS and FTD due to C9orf72 mutations, aiming to uncover mechanisms for new biomarkers and therapeutic targets.
Repurposing a human drug to treat Alzheimer’s disease
This project aims to validate an approved drug that inhibits tau accumulation in Alzheimer's, advancing it towards commercialization as a novel treatment for the disease.
Unlocking cognition by Pulsatile GnRH in Alzheimer's DiseasE
The UPGRADE project aims to enhance cognitive performance in Alzheimer's patients by testing the safety and efficacy of pulsatile GnRH therapy based on promising preclinical results.
Preclinical development of new nucleoside-based drug against leukemia
The project aims to develop Carbacitabin (CAB) as a stable, effective treatment for high-risk leukemia patients through preclinical optimization, safety studies, and commercial preparation.