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Pre-clinical validation of a novel gene therapy for a rare disease in non-human primates: Integrative approach to the CMC scale-up process validation of a gene therapy product for Friedreich Ataxia

Project description

Transforming the fight against Friedreich's Ataxia

Friedreich's Ataxia (FRDA) affects teenagers and young adults with an inherited neurodegenerative disease with devastating consequences. Unfortunately, there are currently no effective treatments available. In this context, the EU-funded FRATAXAV project centres on BTX-101, the leading candidate and a remarkable product that has exhibited the ability to restore neurological and cardiac functions in FRDA mouse models. With a vision to reshape FRDA treatment, the project is poised to bring hope and healing to those afflicted by this rare disease.

Objective

Founded in 2018, Biointaxis is an academic spin-off company established by a team of researchers lead by a woman Scientist from the Research Institute Germans Trias i Pujol (IGTP) benefiting from extensive know-how in the identification of genes, biomarkers and translational mechanisms underlying neurodegenerative disorders. Our leading candidate, BTX-101, is currently the best-in-class product for the treatment of Friedreich’s Ataxia (FRDA), a rare devastating inherited, progressive, neurodegenerative disease typically affecting teenagers and young adults for whom there are no treatments. Our product proves treatment efficacy being able to re-establish the neurological and cardiac functions in two FRDA mice models. The product is patented (WO2019076973A1) becoming the first preclinical product ever to show life-long disease modifying activity both for neurological and cardiac symptoms in FRDA. Biointaxis has a unique know-how, based on years of fundamental research which has resulted in strong collaborations from important research centers including the Research Institute Germans Trias i Pujol (IGTP) affiliated to the Universitat Autònoma de Barcelona, top University in Spain; reference public Hospitals (University Hospital Germans Trias i Pujol in Badalona, The Virgen del Rocío University Hospital in Seville, Valdecilla University Hospital in Cantabria); public administration (Biocat - la Fundació BioRegió de Catalunya) and foundations (La Caixa, Federación de Ataxias de España, EuroAtaxia, STOP_Friedreich Ataxia), etc. BTX-101 has already been capable of attracting investment to Biointaxis with a €1M seed round in 2022. After sound pre-clinical and clinical validation completed in the next three years, BTX-101 will become also capable of attracting more interest from pharma customers for further validation and commercialization. Ultimately, Biointaxis aims to change the paradigm in Friedreich's ataxia and to defeat rare diseases to restore patient's health.

Fields of science (EuroSciVoc)

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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

BIOINTAXIS SL
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
CARRETERA DE CAN RUTI CAMI DE LES ESCOLES S/N
08916 Badalona
Spain

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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
Este Cataluña Barcelona
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Links
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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