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

Project description

In situ drug delivery to the brain for CNS diseases

Neurodegenerative disorders affect millions of individuals worldwide and also have a tremendous impact on their families. Treatments are limited due to the inability of therapeutics to cross the blood–brain barrier and reach the causative cell population in the brain. To address this problem, the EU-funded iProg project is developing a clinically validated platform that can deliver drugs locally within the brain with minimal systemic exposure. Using this device, researchers will prepare for a clinical trial to deliver a novel neurotrophic therapy against frontotemporal degeneration, a type of dementia caused by mutations in the progranulin gene. Results will have important implications for the treatment of neurodegenerative disorders.


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.

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Net EU contribution
€ 50 000,00

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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Östra Sverige Stockholm Stockholms län
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Total cost
€ 71 429,00