Objective
REGENERA will assess the technical and commercial feasibility of a novel and optimised C-MANF peptide as a superior treatment for amyotrophic lateral sclerosis (ALS). ALS is a fatal neurodegenerative disease affecting 450,000 people worldwide where motoneurons (MNs) selectively degenerate in the brain and spinal cord. ALS is characterised by muscle deterioration that rapidly leads to disability and culminates in death 3-5 years after diagnosis. Unfortunately, there is no cure for ALS and current treatments only marginally slow down its progression. Moreover, promising neurotrophic factors (NTFs) with neuroprotective activity show insufficient efficiency, are unable to reach the brain tissue, and have highly invasive administration routes (i.e. brain injections and intrathecal) and high production costs. As a result, all NTFs clinical trials have failed.
Ass. Prof. Voutilainen has discovered C-MANF, a novel peptide which, in contrast to classical NTFs, (i) has protective and restorative effects on motoneurons, (ii) penetrates the blood-brain barrier (BBB), (iii) can be subcutaneously administered, and (iv) can be inexpensively produced. Within REGENERA, we will assess whether C-MANF is feasible as an early-therapy option for ALS. Firstly, we will analyse the pharmacokinetic properties and efficacy of C-MANF in ALS animal models and in human MNS. Subsequently, commercial feasibility will be determined by verifying IP position and strategy, performing in-depth market and competitor analyses, and finally consolidating these into a business case to establish the best path to commercialisation. Successful commercialisation of C-MANF could reduce the profound socioeconomic burden of ALS, provide an early-therapy option to delay disease progression and thus extend and improve the patients’ lives, and provide the pharmaceutical industry with a novel therapeutic that can potentially be used for other neurodegenerative diseases.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- natural sciences biological sciences biochemistry biomolecules proteins
- medical and health sciences basic medicine neurology amyotrophic lateral sclerosis
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Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.1.1 - European Research Council (ERC)
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Topic(s)
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Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) ERC-2022-POC1
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
00014 HELSINGIN YLIOPISTO
Finland
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