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DPR-VAX SIGNED

Dipeptide-Repeat Vaccine to prevent ALS and FTD in C9orf72 mutation carriers

Total Cost €

0

EC-Contrib. €

0

Partnership

0

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 DPR-VAX project word cloud

Explore the words cloud of the DPR-VAX project. It provides you a very rough idea of what is the project "DPR-VAX" about.

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Project "DPR-VAX" data sheet

The following table provides information about the project.

Coordinator
DEUTSCHES ZENTRUM FUR NEURODEGENERATIVE ERKRANKUNGEN EV 

Organization address
address: SIGMUND FREUD STRASSE 27
city: BONN
postcode: 53127
website: www.dzne.de

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Germany [DE]
 Total cost 0 €
 EC max contribution 150˙000 € (0%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2019-PoC
 Funding Scheme ERC-POC-LS
 Starting year 2020
 Duration (year-month-day) from 2020-03-01   to  2021-08-31

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    DEUTSCHES ZENTRUM FUR NEURODEGENERATIVE ERKRANKUNGEN EV DE (BONN) coordinator 150˙000.00

Map

 Project objective

Neurodegenerative diseases are triggered by protein aggregation in the CNS, but developing therapies has been challenging. Since key disease mechanisms change during disease progression, halting further neuron loss has not been achieved and may not even improve the symptoms and survival. Causal therapy is most realistic for monogenic disease variants. About 10% of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) cases are caused by repeat expansion in the C9orf72 gene. We have shown that the expanded repeat is translated into multiple aggregating dipeptide repeat (DPR) proteins. Poly-GA, the most abundant DPR species, is a key driver of downstream pathology. A poly-GA vaccine reduces aggregates and almost completely prevents motor deficits in our mouse model. With additional validation and safety experiment we will generate a pharma-ready data package for rapid preclinical and clinical devel-opment of the vaccine as first-in-class drug for prevention of ALS and FTD in C9orf72 carriers. The parallel development of suitable biomarkers to show clearance of DPR aggregates in vivo will greatly accelerate clinical trials. Thus, we will optimize our published immunoassays to allow more sensitive detection of DPRs in patient CSF. Moreover, we will develop our patented DPR monoclonal antibodies into a PET tracer. The solid data package is complemented by internal and external expertise in business development and regulatory affairs to shape an attractive business proposition and create value. ERC-PoC funding will allow us to present the opportunity to potential partners in large pharmaceutical companies and reputable venture capitalists for licensing or spin-off generation. Another proven strategy in the rare disease space would be collaboration with patient organizations to fund initial clinical trials. Preventing fatal ALS and FTD using vaccination in C9orf72 mutation carriers would be cost-effective for society and a blessing for affected families.

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The information about "DPR-VAX" are provided by the European Opendata Portal: CORDIS opendata.

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lastchecktime (2022-08-14 12:18:33) correctly updated