RESERCH

About visual restoration gene therapy                       視覚再生遺伝子治療について

VISUAL RESTORATION GENE THERAPY FOR RETINITIS PIGMENTOSA

視覚再生遺伝子治療薬開発について

BACK GROUND: It is said that vision occupies 80% of the information that people obtain from the outside. If we lose your eyesight, our quality of life will suffer considerably. Ophthalmic medicine is progressing day by day, but there are diseases neither treatment nor prevention methods are established yet. Ophthalmologists cannot do anything but just watching the patient go blind with such disease.
In japan, there are over 300,000 visual impairment patients and the social cost of visual impairment is over $ 80 Bn/ year. And the second cause of the adult blindness is a disease called retinitis pigmentosa. And what is worse, this disease is interactable for now.
THERAPUTIC CONCEPT: It is a simple injection into the eyeball and then the AAV vector delivers this chimeric rhodopsin genes to the target cell. Then, the remaining cells become photoreceptive. And the patient will be able to see the world again.
CORE TECHNOLOGY: We can see this light because our rhodopsin is sensing the light. There are 2 types of rhodopsin, animal rhodopsin and microbial rhodopsin. We hybridized these two types of rhodopsin to create chimeric rhodopsin optimized for visual restoration. This chimeric rhodopsin overcame the weakness of the conventional rhodopsins and it keeps working alone with high sensitivity.

現在、日本の成人視覚障害の原因の第2位を占め、未だ有効な治療法のない指定難病である網膜色素変性症を対象に、慶應義塾大学医学部光生物学研究室の研究成果をもとに、光センサータンパク質であるキメラロドプシンを用いて、視覚再生遺伝子治療薬の開発を行います。キメラロドプシンは動物型と微生物型のロドプシンの特徴を兼ね備える、視覚再生に最適化されたロドプシンです(特許出願中)。同じく研究開発が進められている人工網膜やiPS由来視細胞移植に比べ、大きな手術の必要がなく、眼内に一度治療遺伝子を注射するだけで、半永久的な視覚再生効果が期待できるのが大きな特徴です。
現在、動物実験での有効性が確認できており、今後オーファンドラッグとしての強みを生かした開発で2020年治験開始、2026年上市を目指します。

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  2. About visual restoration gene therapy                       視覚再生遺伝子治療について

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