ナノスコープ・セラピューティクス、オプトジェネティクス・キャッチテクノロジーのライセンスを取得

ナノスコープの遺伝子治療に組み込まれた革新的な技術は、遺伝性視覚障害の視力回復に希望を与える

ダラス, 2024年9月30日 — ナノスコープ・セラピューティクス株式会社遺伝性網膜疾患（IRD）および黄斑変性に伴う地図状萎縮に対する遺伝子治療を開発している臨床段階のバイオテクノロジー企業である は、本日、マックスプランク協会の技術移転組織であるマックスプランクイノベーションから CatCh 技術のライセンスを取得したと発表しました。マックスプランク生物物理学研究所の研究結果と、マックスプランク教授の先駆的な研究に基づき、この技術は、遺伝子治療における重要な鍵となる重要な遺伝子治療です。 エルンスト・バンベルク 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. サマレンドラ・モハンティ, 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. 治療への応用と臨床試験 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. エルンスト・バンベルクオプトジェネティクス分野の先駆者の一人である、マックス・プランク生物物理学研究所の研究者と彼の同僚は、チャネルロドプシン2の変異体で特性が改善されたCatChを開発した。CatChは、反応速度の高速化や青色光に対する感度の向上など、大きな利点がある。 ナノスコープ・セラピューティクス社について ナノスコープ・セラピューティクスは、視覚障害や失明を引き起こす網膜変性疾患を治療するための、変異に依存しない遺伝子治療を開発している臨床段階のバイオ医薬品会社です。同社は、 最近発表された100週間のデータによると、 米国における網膜色素変性症に対するRESTORE第2b/3相多施設ランダム化二重盲検偽対照臨床試験（NCT04945772）。同社は最近、シュタルガルト病患者を対象とした MCO-010 療法の第 2 相 STARLIGHT 試験も完了しました (NCT05417126）。MCO-010 は、RP とシュタルガルト病の両方に対して FDA のファストトラック指定と FDA の希少疾病用医薬品指定を受けています。前臨床資産には、AMD に続発する GA に対する非ウイルス性レーザー送達 MCO-020 遺伝子治療が含まれます。 投資家連絡先:
アルゴットパートナーズ
(212) 600-1902
PR@nanostherapeutics.com 出典 ナノスコープ・セラピューティクス