{"id":8289,"date":"2024-12-19T10:44:57","date_gmt":"2024-12-19T09:44:57","guid":{"rendered":"https:\/\/www.iqac.csic.es\/una-nova-terapia-obre-cami-en-el-tractament-de-la-malaltia-de-fabry\/"},"modified":"2026-06-16T10:47:28","modified_gmt":"2026-06-16T08:47:28","slug":"una-nova-terapia-obre-cami-en-el-tractament-de-la-malaltia-de-fabry","status":"publish","type":"post","link":"https:\/\/www.iqac.csic.es\/ca\/una-nova-terapia-obre-cami-en-el-tractament-de-la-malaltia-de-fabry\/","title":{"rendered":"Una nova ter\u00e0pia obre cam\u00ed en el tractament de la malaltia de Fabry"},"content":{"rendered":"<p>Un equip de recerca internacional liderat per l&#8217;<strong>Institut de Ci\u00e8ncia de Materials de Barcelona (ICMAB-CSIC)<\/strong> i <strong>CIBER-BBN<\/strong>, amb col\u00b7laboraci\u00f3 de l&#8217;<strong>Institut de Qu\u00edmica Avan\u00e7ada de Catalunya (IQAC-CSIC)<\/strong>, ha desenvolupat una nova ter\u00e0pia basada en nanotecnologia anomenada nanoGLA per al tractament de la malaltia de Fabry. La nova soluci\u00f3 terap\u00e8utica ha demostrat una efic\u00e0cia notable en estudis precl\u00ednics. L\u2019estudi s\u2019ha publicat aquest desembre a la revista <a href=\"https:\/\/www.science.org\/doi\/10.1126\/sciadv.adq4738\" target=\"_blank\" rel=\"noopener\"><em>Science Advances<\/em><\/a> en acc\u00e9s obert.<\/p>\n<p>La malaltia de Fabry \u00e9s una malaltia rara d\u2019origen gen\u00e8tic causada pel d\u00e8ficit de l\u2019enzim GLA (alfa-galactosidasa A) que provoca l\u2019acumulaci\u00f3 de substrats greixosos (majorit\u00e0riament globotriaosilceramida o Gb3) a les c\u00e8l\u00b7lules i t\u00e9 greus repercussions a diferents \u00f2rgans. La ter\u00e0pia nanoGLA, basada en l\u2019\u00fas de nanoliposomes dirigits per p\u00e8ptids, permet fer arribar de manera efectiva l\u2019enzim deficitari GLA, encapsulat en els nanoliposomes, als \u00f2rgans m\u00e9s afectats per aquesta malaltia. Els investigadors han aconseguit fabricar la nanoGLA\u00a0 amb la qualitat i la quantitat necess\u00e0ries per al testatge precl\u00ednic, i per a poder entrar a les fases cl\u00edniques.<\/p>\n<p>En estudis amb models de ratolins amb la malaltia de Fabry, la nanoGLA ha demostrat una efic\u00e0cia millorada en relaci\u00f3 a les ter\u00e0pies que utilitzen l\u2019enzim sense encapsular, mostrant efectivitat en els \u00f2rgans afectats, i fins i tot al cervell, una fita clau que les ter\u00e0pies actuals no aconsegueixen. Aquests resultats ressalten el potencial de la nanoGLA per abordar tant les manifestacions sist\u00e8miques com les cerebrovasculars de la malaltia de Fabry.<\/p>\n<p>Com a reconeixement a la import\u00e0ncia d\u2019aquesta innovaci\u00f3, l\u2019Ag\u00e8ncia Europea de Medicaments va atorgar al 2021 a la nanoGLA la designaci\u00f3 de medicament orfe (Orphan Medicinal Product Designation), un pas decisiu per impulsar-ne el desenvolupament.<\/p>\n<p>Aquest aven\u00e7 ha estat possible gr\u00e0cies a la col\u00b7laboraci\u00f3 entre investigadors de diverses institucions internacionals, incloent-hi l\u2019Institut de Ci\u00e8ncia de Materials de Barcelona (ICMAB-CSIC), el CIBER-BBN, el Vall d\u2019Hebron Institut de Recerca (VHIR), empreses com Nanomol Technologies SL i Leanbio SL, l\u2019Institut de Qu\u00edmica Avan\u00e7ada de Catalunya (IQAC-CSIC), l\u2019Institut de Biotecnologia i Biomedicina (IBB-UAB) i col\u00b7laboradors internacionals com el Joanneum Research-Institute for Biomedical Research and Technologies (HEALTH) (\u00c0ustria), el Technion-Israel Institute of Technology (Israel), el Guangdong-Technion Israel Institute of Technology (Xina), Aarhus University (Dinamarca) i Labcorp Drug Development (UK).<\/p>\n<p><strong>Elisabet Gonz\u00e1lez<\/strong>, investigadora de l\u2019ICMAB i una de les principals autores de l\u2019article explica \u201cla nova formulaci\u00f3 nanoGLA representa una oportunitat prometedora per als pacients amb malaltia de Fabry, especialment en abordar les manifestacions neurol\u00f2giques de la malaltia, una limitaci\u00f3 que les ter\u00e0pies actuals no poden superar. El nostre objectiu \u00e9s desenvolupar tractaments m\u00e9s segurs i efectius aprofitant el potencial de la nanotecnologia.\u201d<\/p>\n<p>Aquests \u00a0resultats, obtinguts en el marc del projecte Europeu Smart4Fabry, finan\u00e7at pel programa de recerca i innovaci\u00f3 Horizon 2020 de la Uni\u00f3 Europea, han donat llum verda a la continuaci\u00f3 del desenvolupament farmac\u00e8utic de la nanoGLA cap a les fases cl\u00edniques amb pacients humans. La Comissi\u00f3 Europea, a trav\u00e9s dels projectes EU Phoenix i Nano4Rare, ha atorgat el finan\u00e7ament necessari per completar la fase precl\u00ednica i aconseguir l\u2019aprovaci\u00f3 per iniciar la fase cl\u00ednica.<\/p>\n<p style=\"text-align: right;\"><strong>ICMAB-CSIC Comunicaci\u00f3<\/strong><\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p style=\"font-size: 75%;\"><strong><a href=\"https:\/\/www.science.org\/doi\/10.1126\/sciadv.adq4738\" target=\"_blank\" rel=\"noopener\">Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease<\/a>.<\/strong> Judit Tomsen-Melero, Marc Molt\u00f3-Abad, Josep Merlo-Mas, Zamira V. D\u00edaz-Riascos, Edgar Crist\u00f3bal-Lecina, Andreu Soldevila, Thomas Altendorfer-Kroath, Dganit Danino, Inbal Ionita, Jan Skov Pedersen, Lyndsey Snelling, Hazel Clay, Aida Carre\u00f1o, Jos\u00e9 L. Corchero, Daniel Pulido, Josefina Casas, Jaume Veciana, Sim\u00f3 Schwartz Jr., Santi Sala, Albert Font, Thomas Birngruber, Miriam Royo, Alba C\u00f3rdoba, Nora Ventosa, Ibane Abasolo, and Elisabet Gonz\u00e1lez-Mira <em>Science Advances<\/em> 13 Dec 2024, Vol 10, Issue 50, <a href=\"https:\/\/www.science.org\/doi\/10.1126\/sciadv.adq4738\">DOI: 10.1126\/sciadv.adq4738<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Un equip de recerca internacional liderat per l&#8217;Institut de Ci\u00e8ncia de Materials de Barcelona (ICMAB-CSIC) i CIBER-BBN, amb col\u00b7laboraci\u00f3 de l&#8217;Institut de Qu\u00edmica Avan\u00e7ada de Catalunya (IQAC-CSIC), ha desenvolupat una nova ter\u00e0pia basada en nanotecnologia anomenada nanoGLA per al tractament de la malaltia de Fabry. La nova soluci\u00f3 terap\u00e8utica ha demostrat una efic\u00e0cia notable en<a class=\"more-link\" href=\"https:\/\/www.iqac.csic.es\/ca\/una-nova-terapia-obre-cami-en-el-tractament-de-la-malaltia-de-fabry\/\">Continue reading <span class=\"screen-reader-text\">\u00abUna nova ter\u00e0pia obre cam\u00ed en el tractament de la malaltia de Fabry\u00bb<\/span><\/a><\/p>\n","protected":false},"author":3,"featured_media":4498,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[73],"tags":[],"class_list":["post-8289","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","entry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.7 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Una nova ter\u00e0pia obre cam\u00ed en el tractament de la malaltia de Fabry | IQAC - Institut de Qu\u00edmica Avan\u00e7ada de Catalunya - CSIC<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.iqac.csic.es\/una-nueva-terapia-abre-camino-en-el-tratamiento-de-la-enfermedad-de-fabry\/\" \/>\n<meta property=\"og:locale\" content=\"ca_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Una nova ter\u00e0pia obre cam\u00ed en el tractament de la malaltia de Fabry | IQAC - Institut de Qu\u00edmica Avan\u00e7ada de Catalunya - CSIC\" \/>\n<meta property=\"og:description\" content=\"Un equip de recerca internacional liderat per l&#8217;Institut de Ci\u00e8ncia de Materials de Barcelona (ICMAB-CSIC) i CIBER-BBN, amb col\u00b7laboraci\u00f3 de l&#8217;Institut de Qu\u00edmica Avan\u00e7ada de Catalunya (IQAC-CSIC), ha desenvolupat una nova ter\u00e0pia basada en nanotecnologia anomenada nanoGLA per al tractament de la malaltia de Fabry. 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La nova soluci\u00f3 terap\u00e8utica ha demostrat una efic\u00e0cia notable enContinue reading \"Una nova ter\u00e0pia obre cam\u00ed en el tractament de la malaltia de Fabry\"","og_url":"https:\/\/www.iqac.csic.es\/una-nueva-terapia-abre-camino-en-el-tratamiento-de-la-enfermedad-de-fabry\/","og_site_name":"IQAC - Institut de Qu\u00edmica Avan\u00e7ada de Catalunya - CSIC","article_publisher":"https:\/\/www.facebook.com\/iqac.csic.es\/","article_published_time":"2024-12-19T09:44:57+00:00","article_modified_time":"2026-06-16T08:47:28+00:00","og_image":[{"width":2542,"height":1589,"url":"https:\/\/www.iqac.csic.es\/wp-content\/uploads\/2026\/06\/Article_nanoGLA_fig.jpg","type":"image\/jpeg"}],"author":"Ana Sotres","twitter_card":"summary_large_image","twitter_creator":"@IQAC_CSIC","twitter_site":"@IQAC_CSIC","twitter_misc":{"Escrit per":"Ana Sotres","Temps estimat de lectura":"3 minuts"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/www.iqac.csic.es\/una-nueva-terapia-abre-camino-en-el-tratamiento-de-la-enfermedad-de-fabry\/#article","isPartOf":{"@id":"https:\/\/www.iqac.csic.es\/una-nueva-terapia-abre-camino-en-el-tratamiento-de-la-enfermedad-de-fabry\/"},"author":{"name":"Ana Sotres","@id":"https:\/\/www.iqac.csic.es\/#\/schema\/person\/ac65d00561d3a6d2810128c46771aff7"},"headline":"Una nova ter\u00e0pia obre cam\u00ed en el tractament de la malaltia de Fabry","datePublished":"2024-12-19T09:44:57+00:00","dateModified":"2026-06-16T08:47:28+00:00","mainEntityOfPage":{"@id":"https:\/\/www.iqac.csic.es\/una-nueva-terapia-abre-camino-en-el-tratamiento-de-la-enfermedad-de-fabry\/"},"wordCount":658,"publisher":{"@id":"https:\/\/www.iqac.csic.es\/#organization"},"image":{"@id":"https:\/\/www.iqac.csic.es\/una-nueva-terapia-abre-camino-en-el-tratamiento-de-la-enfermedad-de-fabry\/#primaryimage"},"thumbnailUrl":"https:\/\/www.iqac.csic.es\/wp-content\/uploads\/2026\/06\/Article_nanoGLA_fig.jpg","articleSection":["News"],"inLanguage":"ca"},{"@type":"WebPage","@id":"https:\/\/www.iqac.csic.es\/una-nueva-terapia-abre-camino-en-el-tratamiento-de-la-enfermedad-de-fabry\/","url":"https:\/\/www.iqac.csic.es\/una-nueva-terapia-abre-camino-en-el-tratamiento-de-la-enfermedad-de-fabry\/","name":"Una nova ter\u00e0pia obre cam\u00ed en el tractament de la malaltia de Fabry | IQAC - 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