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Tailored cell-based therapies for frontotemporal dementia and related genetically defined CNS orphan indications

Descrizione del progetto

Somministrazione di farmaci in situ al cervello per le malattie dell’SNC

Le malattie neurodegenerative colpiscono milioni di persone in tutto il mondo e hanno forti ripercussioni sulle loro famiglie. I trattamenti sono limitati poiché le terapie non riescono ad attraversare la barriera ematoencefalica e a raggiungere la popolazione cellulare causa della patologia nel cervello. Per affrontare il problema, il progetto iProg, finanziato dall’UE, sta sviluppando una piattaforma convalidata clinicamente in grado di somministrare farmaci a livello locale all’interno del cervello con un’esposizione sistemica minima. Attraverso il dispositivo, i ricercatori si prepareranno a una sperimentazione clinica per fornire una nuova terapia neurotrofica contro la degenerazione frontotemporale, un tipo di demenza causato da mutazioni del gene progranulina. I risultati del progetto avranno importanti implicazioni per il trattamento delle malattie neurodegenerative.

Obiettivo

Sinfonia Biotherapeutics is a high-tech biotechnology company. Our vision is to become a main driver in technology innovation for CNS medicine, where we lay the frame-work for entirely novel therapeutic concepts and solutions that combat the unmet medical need of severe CNS conditions, ultimately transforming patient lives.
We are developing targeted therapies for genetically defined CNS orphan indications that are built on a proprietary medical device – the Brain Repair Device - that overcome several of the existing challenges associated with the failing clinical development of drug candidates for neurodegenerative diseases today. The BRD is an implantable device that allows for the in situ production of our therapeutic biologics resulting in desired CNS exposure, while the systemic exposure of the therapeutic factor is negligible. We are developing a new generation of tailored biologics for genetically defined disease/patient populations that target the disease locally in the brain. With our therapies we are thus reaching the right patient, with the right treatment, in an efficacious and safe manner.
Through the SME Instrument, we will focus on further developing iProg, a novel neurotrophic therapy that restores progranulin levels in the brain of patients that suffer from frontotemporal degeneration due to mutations in the PGRN gene (FTD/PGRN), which results in lower progranulin production and is disease-causative. We will use the SME Instrument funding (Phase 1&2) to prepare and conduct PhaseI/IIa studies to explore the safety and detect the first signals of efficacy of iProg. If successful, the project will set new standards on the biologics for neurodegenerative disease market, paving a way to growth and new potential partnerships for Sinfonia. Our disease-modifying therapies have the potential to leverage an important framework for therapeutic development in neurodegenerative disorders in general and thereby to become real game changers in CNS medicine.

Invito a presentare proposte

H2020-EIC-SMEInst-2018-2020

Vedi altri progetti per questo bando

Bando secondario

H2020-SMEInst-2018-2020-1

Meccanismo di finanziamento

SME-1 - SME instrument phase 1

Coordinatore

SINFONIA BIOTHERAPEUTICS AB
Contribution nette de l'UE
€ 50 000,00
Indirizzo
HALSOVAGEN 7, NOVUM
141 57 HUDDINGE
Svezia

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PMI

L’organizzazione si è definita una PMI (piccola e media impresa) al momento della firma dell’accordo di sovvenzione.

Regione
Östra Sverige Stockholm Stockholms län
Tipo di attività
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Collegamenti
Costo totale
€ 71 429,00