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

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

Reporting period: 2018-12-01 to 2019-05-31

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.
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).
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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