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

Informations projet

iProg

N° de convention de subvention: 837501

DOI

10.3030/837501

Projet clôturé

Date de signature de la CE 19 Novembre 2018

Date de début 1 Decembre 2018

Date de fin 31 Mai 2019

Financé au titre de

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Coût total

€ 71 429,00

Contribution de l’UE

€ 50 000,00

Coordonné par

SINFONIA BIOTHERAPEUTICS AB
Sweden

Periodic Reporting for period 1 - iProg (Tailored cell-based therapies for frontotemporal dementia and related genetically defined CNS orphan indications)

Période du rapport: 2018-12-01 au 2019-05-31

Résumé du contexte et des objectifs généraux du projet

Neurodegenerative diseases represent a major threat to human health. They are devastating, often lethal conditions that affect more than 45 million people worldwide. With the aging of the general population, the prevalence of these disorders is expected to rise dramatically in the next few decades, making them one of the largest public health and social care challenges facing us today. Examples of neurodegenera-tive diseases include Alzheimer's disease (AD), PD, amyotrophic lateral sclerosis (ALS), FTD and Lewy body dementia (LBD). Currently, there is no treatment that can prevent the onset or slow progres-sive worsening of neurodegenerative disorders. What must be noted is that these diseases not only take a tremendous toll on the affected individuals but also on their families and society. It is the patient’s family who commonly take the role of caregivers and bear the high costs of care.

Travail effectué depuis le début du projet jusqu’à la fin de la période considérée dans le rapport et principaux résultats atteints jusqu’à présent

This project has performed the feasibility assessment of iProg therapy – a novel neurotrophic therapy that restores progranulin levels in the brain, a secreted pleiotropic growth factor implicated in Frontotemporal dementia (FTD) due to mutations in the progranulin encoding gene (FTD/PGRN), which result in partial loss of Progranulin signalling and impaired lysosomal function. iProg is based on Sinfonia's proprietary medical device – the Brain Repair Device (BRD).

Progrès au-delà de l’état des connaissances et impact potentiel prévu (y compris l’impact socio-économique et les conséquences sociétales plus larges du projet jusqu’à présent)

Sinfonia is developing tailored, targeted therapies for CNS orphan and genetically defined indications that are built on a proprietary medical device – the Brain Repair Device - that overcome the existing challenges associated with the failing clinical development of drug candidates for neurodegenerative diseases today. We are developing novel disease-modifying therapies:
• for the right patient: by focusing on genetically defined diseases/patient populations, we target a known cause of disease in a well-defined patient population, giving us a superior opportunity to tailor the therapy with regard to therapeutic target, dose, treatment duration, and disease stage.
• with the right therapeutic mechanism: our therapies directly target the genetically defined causative pathway of the indication.
• with the right and safe treatment: our therapies are built on the BRD technology, a clinically validated therapeutic platform that allows to target the disease locally at the site needed within the brain, with multiple classes of and also combination of biologics, securing a superior brain exposure whilst keeping the systemic exposure negligible.

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