Nanoscope Therapeutics obtiene la licencia de la tecnología optogenética CatCh

La tecnología innovadora incorporada en la terapia genética de Nanoscope ofrece esperanza para restaurar la visión en discapacidades visuales de origen genético

DALLAS, 30 de septiembre de 2024 — Nanoscopio Therapeutics Inc., una empresa de biotecnología en fase clínica que desarrolla terapias genéticas para enfermedades hereditarias de la retina (ERH) y atrofia geográfica secundaria a la degeneración macular, anunció hoy que la empresa ha adquirido una licencia para la tecnología CatCh de Max Planck Innovation, la organización de transferencia de tecnología de la Sociedad Max Planck. Basándose en los resultados de la investigación del Instituto Max Planck de Biofísica y en el trabajo pionero del profesor. Ernesto Bamberg in the field of optogenetics, this patented technology enhances the light sensitivity of a multi-characteristic opsin (MCO-010, a tri-protein fusion complex including CatCh) developed by Nanoscope as a gene therapy to restore vision in patients suffering from genetically caused visual impairments. Nanoscope’s groundbreaking MCO platform is the first optogenetic approach to combine multiple light-sensitive components, the sum of which produces a fusion protein that is sensitive across the visible spectrum at ambient light levels with fast kinetics. Nanoscope has already successfully completed several clinical studies on MCO-010 for the two leading inherited retinal degenerative diseases: retinitis pigmentosa (RP) and Stargardt disease. The company now plans to seek approval for the therapy and expand to broad therapeutic indications. “We are excited to see the enhanced benefits of the MCO platform, which incorporates the CatCh technology from the Max Planck Institute for Biophysics, and offers new hope for restoring vision in those suffering from severe degenerative retinal conditions,” said Dr. Samarendra Mohanty, Co-Founder & President of Nanoscope Therapeutics. “Our agreement with Max Planck Innovation allows us to transform groundbreaking scientific discoveries into effective therapeutic solutions. With efficient viral delivery of the most complex bioengineered non-mammalian MCO-010 fusion protein to treat severe degenerative retinal conditions caused by many different genetic mutations, we are on the cusp of treating patients with high unmet needs and sustainably improving their quality of life.” Dr. Mareike Göritz, Patent and Licensing manager at Max Planck Innovation, added: “The advanced properties of CatCh in combination with Nanoscope’s proprietary technology make this a very promising approach for gene therapy treatment of retinitis-related visual impairments. We are excited to follow the further development and would be delighted if patients were ultimately to benefit from this innovative approach.” Blindness and visual impairments caused by genetic eye diseases such as retinitis pigmentosa, Stargardt disease, and age-related macular degeneration pose a significant medical challenge worldwide. These diseases often lead to a progressive loss of vision and have a substantial impact on the quality of life of those affected. As current treatment options are limited, innovative approaches like the MCO platform now offer new hope. By its ability to partially restore vision through gene therapy, Nanoscope’s technology platform could represent a significant breakthrough in the treatment of these serious eye diseases. Aplicación terapéutica y pruebas clínicas The now-licensed CatCh technology has been integrated by Nanoscope Therapeutics as one of three subunits of the MCO-010 fusion protein, each of which contributes complementary light-activated properties, that together result in a treatment with the potential to restore vision in everyday settings. Nanoscope’s novel MCO-010 therapeutic approach has been successfully tested in multiple clinical studies for RP and Stargardt disease. Additionally, the MCO platform is already being tested and shown to be effective in non-human primates with geographic atrophies (GA) secondary to advanced age-related macular degeneration (AMD). The further development and approval pathway for MCO-010 is currently being intensively reviewed. Nanoscope Therapeutics aims to obtain approval and is in discussions with the US Food and Drug Administration (FDA) to explore possible accelerated pathways to market. These efforts could help make the innovative therapy available to patients more quickly, who urgently need new treatment options. The CatCh (calcium-transporting channelrhodopsin) technology was developed by Prof. Ernesto Bamberg, uno de los pioneros en el campo de la optogenética, y sus colegas del Instituto Max Planck de Biofísica. CatCh, un mutante de la canalrodopsina-2 con propiedades mejoradas, ofrece ventajas significativas como una cinética más rápida y una mayor sensibilidad a la luz azul. Acerca de Nanoscope Therapeutics Inc. Nanoscope Therapeutics es una empresa biofarmacéutica en fase clínica que desarrolla terapias genéticas independientes de las mutaciones para tratar enfermedades degenerativas de la retina que causan deterioro de la visión y ceguera, para las que hasta la fecha no existe cura. La empresa anunció recientemente los datos de 100 semanas de el ensayo clínico RESTORE de fase 2b/3, multicéntrico, aleatorizado, doble ciego y controlado con placebo en EE. UU. para la retinitis pigmentosa (NCT04945772). La compañía también completó recientemente el ensayo de fase 2 STARLIGHT de la terapia MCO-010 en pacientes con enfermedad de Stargardt (NCT05417126). El MCO-010 ha recibido designaciones de vía rápida de la FDA y de medicamento huérfano de la FDA tanto para la RP como para la enfermedad de Stargardt. Los activos preclínicos incluyen la terapia génica MCO-020 administrada con láser no viral para la atrofia genitourinaria secundaria a la DMAE. Contacto de inversores:
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