{"id":5983,"date":"2025-03-24T15:02:16","date_gmt":"2025-03-24T20:02:16","guid":{"rendered":"https:\/\/nanostherapeutics.com\/?p=5983"},"modified":"2025-07-13T15:10:10","modified_gmt":"2025-07-13T20:10:10","slug":"nanoscope-announces-publication-of-clinical-data-on-vision-restoration-in-retinitis-pigmentosa","status":"publish","type":"post","link":"https:\/\/nanostherapeutics.com\/es\/2025\/03\/24\/nanoscope-announces-publication-of-clinical-data-on-vision-restoration-in-retinitis-pigmentosa\/","title":{"rendered":"Nanoscope anuncia la publicaci\u00f3n de datos cl\u00ednicos sobre la restauraci\u00f3n de la visi\u00f3n en la retinosis pigmentaria"},"content":{"rendered":"
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DALLAS<\/span><\/span>,\u00a024 de marzo de 2025<\/span><\/span> \u2014 Investigadores con\u00a0Nanoscopio Therapeutics Inc.<\/a>, una empresa de biotecnolog\u00eda comprometida con restaurar la visi\u00f3n en pacientes ciegos en entornos del mundo real mediante el desarrollo y la comercializaci\u00f3n de nuevas terapias gen\u00e9ticas para enfermedades degenerativas de la retina, ha anunciado la publicaci\u00f3n de un art\u00edculo,\u00a0\u201cUna opsina sint\u00e9tica restaura la visi\u00f3n en pacientes con degeneraci\u00f3n retiniana grave\u201d.\u201d<\/b><\/a>\u00a0en\u00a0Terapia molecular,\u00a0<\/i>La revista insignia de la Sociedad Estadounidense de Terapia G\u00e9nica y Celular. La publicaci\u00f3n del art\u00edculo representa un gran avance en la monoterapia optogen\u00e9tica independiente de mutaciones para pacientes que padecen enfermedades retinianas hereditarias.<\/p>\n

\u201cNos entusiasman los beneficios terap\u00e9uticos observados en pacientes con retinosis pigmentaria que presentan una p\u00e9rdida progresiva y permanente de fotorreceptores y que no tienen ninguna opci\u00f3n de tratamiento\u201d, dijo el Dr.\u00a0Santosh Mahapatra<\/span>, el investigador principal del estudio y director m\u00e9dico del JPM Rotary Eye Hospital (Odisha) en\u00a0India<\/span>. \u201cEsperamos seguir desarrollando esta tecnolog\u00eda para ayudar a quienes sufren una p\u00e9rdida grave de visi\u00f3n debido a enfermedades degenerativas de la retina.\u201d<\/p>\n

Las enfermedades degenerativas retinianas hereditarias (EDR) son la principal causa de ceguera en adultos en edad laboral a nivel mundial. En etapas avanzadas, la p\u00e9rdida celular hace que la edici\u00f3n o el reemplazo g\u00e9nico sean ineficaces. La optogen\u00e9tica ofrece una oportunidad terap\u00e9utica para restaurar la visi\u00f3n mediante la fotosensibilizaci\u00f3n de las neuronas retinianas restantes. Sin embargo, las opsinas actuales son cin\u00e9ticamente lentas, se activan parcialmente con luz ambiental, no responden a diferentes colores de luz y se dirigen a circuitos celulares retinianos de baja resoluci\u00f3n. Para superar estas limitaciones, el equipo de Nanoscope dise\u00f1\u00f3 una sintopsina compuesta por tres prote\u00ednas no mam\u00edferas mutadas selectivamente para lograr una opsina multicaracter\u00edstica (MCO) de banda ancha.<\/p>\n

\u201cEste art\u00edculo destaca el trabajo excepcional del equipo de Nanoscope en el desarrollo de terapias optogen\u00e9ticas eficaces para pacientes con algunas de las necesidades m\u00e1s urgentes no satisfechas\u201d, dijo el Dr.\u00a0Samarendra Mohanty<\/span>, autor principal del art\u00edculo y presidente de Nanoscope. \u201cLos resultados positivos de nuestro ensayo de fase 1\/2a, descritos en este art\u00edculo, junto con los datos del ensayo controlado aleatorizado en retinosis pigmentaria, representan un importante avance en el tratamiento de las enfermedades retinianas hereditarias\u201d.\u201d<\/p>\n

La sintopsina modificada se empaquet\u00f3 en un vector de terapia g\u00e9nica AAV2 optimizado que se dirige a las c\u00e9lulas bipolares de la retina humana. En un estudio abierto iniciado por un investigador, cuatro pacientes ciegos con retinosis pigmentaria con\u00a0ABCA4<\/i>\u00a0Las variantes recibieron una \u00fanica inyecci\u00f3n intrav\u00edtrea de terapia g\u00e9nica. Las im\u00e1genes no invasivas confirmaron la expresi\u00f3n g\u00e9nica retiniana mediante una prote\u00edna reportera fluorescente. Los pacientes mostraron una mejor\u00eda en la agudeza visual, la discriminaci\u00f3n de formas y la movilidad durante las 52 semanas del estudio. No se observaron problemas de seguridad significativos a pesar de que probablemente sea una de las prote\u00ednas no mam\u00edferas m\u00e1s sint\u00e9ticas jam\u00e1s expresadas en humanos. Este es el primer informe de una monoterapia g\u00e9nica que puede restaurar la visi\u00f3n en pacientes ciegos de forma independiente de las mutaciones, utilizando una plataforma tecnol\u00f3gica optogen\u00e9tica.<\/p>\n

\u201cNuestro estudio representa un hito fundamental en el tratamiento de las degeneraciones retinianas hereditarias, al demostrar que la optogen\u00e9tica puede restaurar con \u00e9xito la visi\u00f3n en pacientes ciegos, independientemente de su mutaci\u00f3n gen\u00e9tica\u201d, dijo el Dr.\u00a0Vinit B. Mahajan<\/span>, coautor correspondiente y profesor y vicedirector de investigaci\u00f3n del Departamento de Oftalmolog\u00eda de la Universidad de Stanford. \u201cLa capacidad de restaurar la visi\u00f3n con una sola inyecci\u00f3n intrav\u00edtrea representa un paso significativo hacia el desarrollo de un tratamiento universal para las enfermedades degenerativas de la retina\u201d.\u201d<\/p>\n

Haga clic aqu\u00ed para acceder al art\u00edculo completo y a la representaci\u00f3n gr\u00e1fica del estudio cl\u00ednico.<\/a><\/p>\n

Acerca de Nanoscope Therapeutics Inc.
<\/b>Nanoscope Therapeutics est\u00e1 desarrollando terapias optogen\u00e9ticas, independientes de las mutaciones y que restauran la visi\u00f3n, para los millones de pacientes con ceguera causada por enfermedades degenerativas de la retina para las que actualmente no existe tratamiento. Tras los resultados positivos al final del estudio RESTORE, ensayo cl\u00ednico multic\u00e9ntrico, aleatorizado, doble ciego y controlado con placebo de fase 2b para la RP (NCT04945772<\/a>), la compa\u00f1\u00eda anunci\u00f3 su plan de iniciar un\u00a0Presentaci\u00f3n de BLA para MCO-010 para tratar RP<\/a>\u00a0en el primer semestre de 2025. La empresa ha completado el ensayo STARLIGHT de fase 2 de la terapia MCO-010 en pacientes con Stargardt (NCT05417126<\/a>) y planea iniciar una\u00a0Ensayo de registro de fase 3<\/a>Tambi\u00e9n en el primer semestre de 2025, MCO-010 ha recibido las designaciones de v\u00eda r\u00e1pida de la FDA y de medicamento hu\u00e9rfano de la FDA tanto para RP como para Stargardt. Los programas precl\u00ednicos incluyen un activo MCO-020 de administraci\u00f3n l\u00e1ser no viral, listo para IND, para AG, as\u00ed como un activo AAV para la amaurosis cong\u00e9nita de Leber, en estudios que facilitan IND.<\/p>\n

Contacto de inversores:<\/b>
Socios Argot
212-600-1902
PR@nanostherapeutics.com<\/a><\/p>\n

FUENTE Terap\u00e9utica del Nanoscopio<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"

DALLAS,\u00a0March 24, 2025 \u2014\u00a0Researchers with\u00a0Nanoscope Therapeutics Inc., a biotechnology company committed to restoring vision in blind patients in real-world settings by developing and commercializing novel gene therapies for retinal degenerative diseases, have announced the publication of a paper,\u00a0\u201cA synthetic opsin restores vision in patients with severe retinal degeneration,\u201d\u00a0in\u00a0Molecular Therapy,\u00a0the flagship journal of the American Society […]<\/p>\n","protected":false},"author":6,"featured_media":5986,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_exactmetrics_skip_tracking":false,"_exactmetrics_sitenote_active":false,"_exactmetrics_sitenote_note":"","_exactmetrics_sitenote_category":0,"footnotes":"","_links_to":"","_links_to_target":""},"categories":[13,8],"tags":[],"class_list":["post-5983","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-home-page","category-nanoscope-press-release"],"yoast_head":"\nNanoscope Announces Publication of Clinical Data on Vision Restoration in Retinitis Pigmentosa - Nanoscope Therapeutics<\/title>\n<meta name=\"description\" content=\"Our multi-characteristic opsin (MCO) platform is the first to demonstrate an ability to restore vision to retinal disease patients with severe vision loss in clincial trials. 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