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Development of life changing drugs for patients suffering from severe and fatal neurological disorders

Project description

Life-changing drug development for neurological disorders

Disorders of the central nervous system can often be severe or fatal and they affect many people. Treatment options are typically limited for these patients. The EU-funded RespirOndine project, in collaboration with experts from the Congenital Central Hypoventilation Syndrome (CCHS) patient organisation, aims to develop technology and therapeutic solutions to improve treatments for these patients. Firstly, they plan to focus on CCHS, which frequently leads to severe hypoventilation episodes. They have identified and optimised an innovative compound that can be used to develop a groundbreaking drug that could potentially save lives by eliminating the need for bulky machinery.

Objective

AtmosR is a start-up whose mission is to develop innovative therapeutic solutions for patients suffering from severe and often fatal disorders of the central nervous system.

Our company has established a technology in order to develop treatments for aggregate pathologies (such as ALS, Kennedy disease, Multisystem Atrophy, Parkinson, Huntington...) which represent a major health problem considering the high number of patients worldwide and the high unmet medical need.

The first disorder targeted by AtmosR is Congenital Central Hypoventilation Syndrome (CCHS), a rare genetic syndrome which presents as its main symptom severe periods of hypoventilation. Patients are kept alive thanks to mechanical ventilation administered through a tracheostomy. Our goal is to develop a drug that will be life changing for patients and their families, liberating them from the machine, lifting the vital risk and allowing them to live independently.

Our team consists of multidisciplinary profiles with experience in both drug development, particularly in the field of rare diseases, and in business management. As a spin-off of the french CCHS patient organization, we work in close cooperation with the international academic experts in CCHS, especially in France, Italy and Israël.

We have identified a Hit compound ATS-003 that is active in both in vitro and in vivo CCHS robust models and we have optimized the compound thanks to a chemistry program in order to improve its efficiency and lower its toxicity for a better long term use tolerance. We are starting regulatory preclinical work in 2023 with this new Lead compound and have planned the first clinical trials by mid 2025 and an early drug access for patients in 2027.

In addition to CCHS, we have identified several disorders with similar protein aggregation mechanisms. We plan to test and develop our Lead compound for other more common disorders and the purpose of this project is to idententify the most promising one for our Lead.

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Keywords

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Programme(s)

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Topic(s)

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.

HORIZON-CSA - HORIZON Coordination and Support Actions

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Call for proposal

Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.

(opens in new window) HORIZON-EIE-2022-SCALEUP-02

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Coordinator

ATMOSR
Net EU contribution

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.

€ 75 000,00
Address
10 IMPASSE DES LOTIERS
13710 Fuveau
France

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Provence-Alpes-Côte d’Azur Provence-Alpes-Côte d’Azur Bouches-du-Rhône
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
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Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

No data
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