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

Información del proyecto

iProg

Identificador del acuerdo de subvención: 837501

DOI

10.3030/837501

Proyecto cerrado

Fecha de la firma de la CE 19 Noviembre 2018

Fecha de inicio 1 Diciembre 2018

Fecha de finalización 31 Mayo 2019

Financiado con arreglo a

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Coste total

€ 71 429,00

Aportación de la UE

€ 50 000,00

Coordinado por

SINFONIA BIOTHERAPEUTICS AB
Sweden

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

Período documentado: 2018-12-01 hasta 2019-05-31

Resumen del contexto y de los objetivos generales del proyecto

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.

Trabajo realizado desde el comienzo del proyecto hasta el final del período abarcado por el informe y los principales resultados hasta la fecha

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).

Avances que van más allá del estado de la técnica e impacto potencial esperado (incluida la repercusión socioeconómica y las implicaciones sociales más amplias del proyecto hasta la fecha)

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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