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Pre-clinical validation of a novel gene therapy for a rare disease in non-human...

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 Friedreic 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 identifi... ver más

30/06/2024

BIOINTAXIS

75K€

Presupuesto del proyecto: 75K€

Líder del proyecto

BIOINTAXIS La investigacion cientifica y tecnica que conduzca al descubrimiento, desarrollo y comercializacion de productos terapeuticos, tratamientos...

TRL 5-6 | 592K€

Financiación concedida El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2024-06-30

HORIZON-EIE-2022-SCALEUP-02-02: Women TechEU ExpectedOutcome:Project results are expected to contribute to the following expected outcomes:

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Cerrada hace 2 años

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BIOINTAXIS

75.00K€ | Lider

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Duración del proyecto: 12 meses Fecha Inicio: 2023-06-05
Fecha Fin: 2024-06-30

Líder del proyecto

BIOINTAXIS La investigacion cientifica y tecnica que conduzca al descubrimiento, desarrollo y comercializacion de productos terapeuticos, tratamientos...

TRL 5-6 | 592K€

Presupuesto del proyecto 75K€

Descripción del proyecto 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.

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