Publications & Grants

The latest publications and competitive R&D grants related to our research work, on advanced RNA treatments against genetic diseases,can be consulted online on this page.

A two-pronged approach to myotonic dystrophy type 1
Spain’s ARTHEx Biotech has developed a dual-mechanism approach to treating the most common adult-onset muscular dystrophy, myotonic dystrophy type 1, setting it apart in a burgeoning blockbuster market.
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AntimiR treatment corrects myotonic dystrophy primary cell defects across several CTG repeat expansions with a dual mechanism of action
This study evaluated therapeutic antimiRs in primary myoblasts from patients with myotonic dystrophy type 1 (DM1). DM1 results from unstable CTG repeat expansions in the DMPK gene, leading to variable clinical manifesta- tions by depleting muscleblind-like splicing regulator protein MBNL1.
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Lead antimiR-23b drug rescues cell phenotypes in a range of myotonic dystrophy genetic backgrounds
Judy Walker, Estefanía Cerro-Herreros, Judit Núñez-Manchón, Neia Naldaiz-Gastesi, Marc Carrascosa, Sàez, Andrea García-Rey, Irene González-Martínez, Kevin Moreno, Javier Poyatos-García, Juan J Vilchez, Adolfo López de Munain, Mònica Suelves, Gisela Nogales-Gadea, Beatriz Llamusí and Rubén Artero. 2022 MDF Annual Conference. 9th-10th September. San Diego (California)
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New ARTHEx Biotech presentation at the 2022 Myotonic Dystrophy Foundation program "Meet the DM drug developers"
Meet the DM drug developers is a program organized by the Myotonic Dystrophy Foundation where each month biotechnology, pharmaceutical and academic partners, large and small, working on treatments for myotonic dystrophy sit down with the Myotonic Dystrophy Foundations community to share their progress and answer their questions. Watch Arthex's presentation on June 10, 2022, where we explain our updated program for the development of ATX-01.
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Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model
Sarah J. Overby, Estefanía Cerro-Herreros, Irene González-Martínez, Miguel A. Varela, David Seoane-Miraz, Yahya Jad, Richard Raz, Thorleif Møller, Manuel Pérez-Alonso, Matthew J. Wood, Beatriz Llamusí, Rubén Artero. Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model. Molecular Therapy - Nucleic Acids, Volume 27,2022, Pages 1146-1155.
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ARTHEx biotech presentation at the Myotonic Dystrophy Foundation program "Meet the DM drug developers"
Meet the DM drug developers is a program organized by the Myotonic Dystrophy Foundation where each month biotechnology, pharmaceutical and academic partners, large and small, working on treatments and a cure for myotonic dystrophy sit down with the Myotonic Dystrophy Foundations community to share their progress and answer their questions. Watch Arthex's presentation on June 9, 2021, where we explain our program for the development of ATX-01.
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Therapeutic Potential of AntagomiR-23b for Treating Myotonic Dystrophy
Estefanía Cerro-Herreros, Irene González-Martínez, Nerea Moreno-Cervera, Sarah Overby, Manuel Pérez-Alonso, Beatriz Llamusí, Rubén Artero Mol Ther Nucleic Acids. 2020 Sep 4; 21: 837–849.
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Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy
Estefanía Cerro-Herreros, Irene González-Martínez, Nerea Moreno, Jorge Espinosa-Espinosa, Juan M. Fernández-Costa, Anna Colom-Rodrigo, Sarah J. Overby, David Seoane-Miraz, Javier Poyatos-García, Juan J. Vilchez, Adolfo López de Munain, Miguel A. Varela, Matthew J. Wood, Manuel Pérez-Alonso, Beatriz Llamusí, Rubén Artero Mol Ther Nucleic Acids. 2021 Dec 3; 26: 174–191.
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miR-23b and miR-218 silencing increase Muscleblind-like expression and alleviate myotonic dystrophy phenotypes in mammalian models
Estefania Cerro-Herreros, Maria Sabater-Arcis, Juan M. Fernandez-Costa, Nerea Moreno, Manuel Perez-Alonso, Beatriz Llamusi, Ruben Artero Nat Commun. 2018; 9: 2482.
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Instituto Valenciano de Competitividad Empresarial (IVACE). PROYECTOS DE I+D DE PYME (PIDI-CV)

ARTHEx has been awarded funding by GVA-IVACE to support R&D and innovation projects. The approved project, titled "Development of Novel Delivery Systems for Therapeutic Oligonucleotides Targeting Specific Tissues" (“Desarrollo de nuevos sistemas de liberación de oligonucleótidos terapéuticos dirigidos a tejidos diana”) (IMIDTA/2024/22), aims to advance cutting-edge technologies for the precise delivery of oligonucleotide-based therapeutics.

This initiative is part of the R&D Projects for SMEs (PIDI-CV) 2024 program and is co-financed by the European Union through the European Regional Development Fund (Fondos FEDER). The total eligible cost of the project is 169.910 €, of which 70.682,56 € has been granted as a subsidy.

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Proyectos en Colaboración Público Privada 2022

The project “Translational Research on Myotonic Dystrophy for the Development of an RNA Based Advanced Therapy (DISMIOTATX)” with reference CPP2022-009960 is granted by the “Proyectos en Colaboración Público Privada 2022” program funded by MCIN/AEI/10.13039/501100011033 and, as appropriate by the “European Union NextGenerationEU/PRTR”.

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AVI INNTAL L2

ARTHEx receives financial support from the Talent Promotion Program-Line 2 of GVA-AVI for the execution of the project "Development of the pivotal (GLP) and toxicity studies program for an RNA drug" (INNTA2/2023/10). The project is granted with 75.184,18€, with co-financing from the European Union through the European Regional Development Fund (FEDER) 2021-2027.

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AVI INNTAL L3

ARTHEx receives financial support from the Talent Promotion Program-Line 3 of GVA-AVI for the execution of the project "Efficacy studies of an antagomir-23-B in the treatment of spliceopathies" (INNTA3/2023/16). The project is granted with 40.191,75 €, with co-financing from the European Union through the European Regional Development Fund (FEDER) 2021-2027.

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Conselleria de Economía Sostenible, Sectores Productivos, Comercio y Trabajo

Arthex has been a beneficiary of a grant to support the external promotion of the Comunitat Valenciana in the framework of the covid-19 crisis, for the 2022 fiscal year from the Conselleria de Economía Sostenible, Sectores Productivos, Comercio y Trabajo (grant reference INTPRM/2022/764).

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Proyectos I+D+i en «Líneas Estratégicas» 2022

The project “New fly and 3D cell tools for the development of therapeutic oligonucleotides” with reference PLEC2022-009367 is granted by the “Proyectos I+D+i en «Líneas Estratégicas” program funded by MCIN/AEI/10.13039/501100011033 and, as appropriate by the “European Union NextGenerationEU/PRTR”.

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Proyectos en Colaboración Público Privada 2021

The project “Development of a first-in-class therapy for the treatment of osteoarthritis (OA) based on microRNA inhibitor oligonucleotides” with reference CPP2021-008792 is granted by the “Proyectos en Colaboración Público Privada 2021” program funded by MCIN/AEI/10.13039/501100011033 and, as appropriate by the “European Union NextGenerationEU/PRTR”.

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FIGHT-DM1

This project has been co-funded by the European Union`s EIC Accelerator program with a 2.5M€ grant, under the Grant Agreement Nº 190181217.

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Innovative SME
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Arthex Biotech is granted as Innovative SME by the Spanish Ministry of Science and Innovation in compliance with the national legislation (Orden ECC/1087/2015, de 5 de junio).
Generalitat Valenciana - Institut Valencià de Finances

ARTHEx has benefited from a financial instrument within the Operational Program of the European Regional Development Fund (FEDER) of the Valencian Community 2014-2020("Venture Capital to Support Business Growth"), managed by the Valencian Institute of Finance (IVF), in collaboration with In Vivo Capital Partners SGEIC, S.A., to support businesses in the Valencian Community with high growth potential.

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CDTI Misiones

ARTHEx is participating in the "OLIGOFASTX: Comprehensive Platform for the Sustainable Development of Oligonucleotide-Based Therapies" project. The general objective of this project is to develop a comprehensive platform for new therapies based on oligonucleotides (AntimiRNAs, siRNAs, and aptamers) that promotes and facilitates the market introduction of new therapies for rare diseases without medical solutions. This project is subsidized by CDTI with the support of the Ministry of Science and Innovation and the European Union's Recovery, Transformation, and Resilience Plan (financed by the European Union-Next Generation EU). The total eligible cost for ARTHEx is €873,439.20. In addition to ARTHEx, the following entities are participating in this project: Sylentis, S.A.U; AptaTargets, S.L.; Aptus Biotech, S.L.; Nostrum Biodiscovery, S.L.; Nanovex Biotechnologies, S.L.; and Biotechnology Development for Industry in Pharmaceutical S.L.U. For more information about the project, please visit:

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Torres Quevedo

ARTHEx is the beneficiary of the Torres Quevedo grant from the “Agencia Estatal de Invetsigación” for the hiring of research personnel. The total eligible cost is €92,489.61. Grant PTQ2020-011110 funded by MCIN/AEI/ 10.13039/501100011033 by“European Union NextGenerationEU/PRTR”.

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IVACE I+D (Valencian Regional Government)

ARTHEx has been granted funding by GVA-IVACE for R&D and innovation projects to carry out the project "In Vitro Efficacy of Oligonucleotides in Different Experimental Disease Models" (IMIDTA/2021/65). The objective of this project is to research the potential of unique antisense RNA oligonucleotides,exclusively developed by the company, as therapeutic targets for various diseases. The total eligible cost is €77,668.81, funded from the 2021 budget,with co-financing from the European Union through the European Regional Development Fund (FEDER).

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AVI INNTAL (Valencian Regional Government)

ARTHEx benefits from the financial support of the Talent Promotion Program - Line 2 of GVA-AVI (Valencian Innovation Agency) for the execution of the project "Development of the CMC program for ATX-01 up to Phase II" (INNTA2/2021/18). The overall objective of the project is to develop the chemistry, manufacturing, and quality program of Arthex Biotech SL necessary to advance our first lead to Phase II clinical trials. The total eligible cost is €173,075.33, funded from the 2021 budget, with co-financing from the European Union through the European Regional Development Fund (FEDER).

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CDTI Neotec

ARTHEx has received support from the Spanish Center for Industrial Technological Development (CDTI) for new business projects of innovative companies (NEOTEC Program), under the State Program for Business R&D and Innovation within the framework of the State Plan for Scientific and Technical Research and Innovation 2017-2020, for the implementation of the project "Antisense Therapy and New Therapeutic Targets for the Treatment of Diseases with Loss of Function of Proteins MBNL1/2" (SNEO-20201136). The total eligible cost is €250,000.

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EIT Health HEADSTART

ARTHEx is supported by the program Headstart of Eit Health (European Union) to execute the project 'ARTHEx-DM1' (2020-HS-0215).. Funding awarded by EIT Health: €50,000

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IVACE CREATEC (Valencian Regional Government)

ARTHEx has received financial support from the CREATEC-CV program of GVA-IVACE for the implementation of the project "Early Preclinical Phases for the Development of an Innovative Drug Against Type 1 Myotonic Dystrophy" (MCBTA/2019/47). The objectives of this project include conducting in vitro toxicity studies to select oligonucleotides with low toxicity and conducting preliminary pharmacokinetic (PK) and pharmacodynamic (PD) studies of these oligonucleotides. The total eligible cost is €55,799.10, funded from the 2019 budget, with co-financing from the European Union through the European Regional Development Fund (FEDER).

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IVACE ASESORA (Valencian Regional Government)

ARTHEx has received financial support from the Specialized Advisory Services for the Growth of Innovative Small Businesses program of GVA-IVACE, ASESORA CV (year 2019) to execute the project "Strategic Diagnosis of Regulatory Affairs and Business Development in the ARTHEx Business Plan" (IMACEI/2019/11). The objective of this project is to carry out a specialized strategic diagnosis in the areas of intellectual property protection and the regulatory framework (FDA). The total eligible cost is €48,883.00, funded from the 2019 budget, with co-financing from the European Union through the European Regional Development Fund (FEDER).

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