216.73.216.231===--UPDATE `0-usmeigara` SET site='',name='',jigyoyoyaku='',sijyo='',gyosyu='',honten='',industry='',sector='',haishi='',keyword='' where code = 'fixx'---select * from `0-usmeigara` WHERE code='fixx'
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銘柄登録／業種登録　関連銘柄専用コメ／リスト／日本株
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遺伝子医薬品会社であるHomology Medicines、Inc。その独自のプラットフォームは、ヌクレアーゼのない遺伝子編集の移動性、遺伝子療法、またはさまざまな遺伝的障害のいずれかを介してin vivoで遺伝子医薬品を生体内で送達するために、そのヒト造血幹細胞由来アデノ関連ウイルスベクター（AAVHSC）を利用するように設計されています。同社はまた、小児患者におけるPKUの治療のためにHMI-103を開発しています。
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「Homology Medicines, Inc., a genetic medicines company, focuses on transforming the lives of patients suffering from rare genetic diseases. Its proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a nuclease-free gene editing mobility, gene therapy or for various genetic disorders. The company's various set of AAVHSCs allows company to precisely target, through a single injection, a range of disease-relevant tissues, including the liver, central nervous system, peripheral nervous system, blood-brain-barrier, bone marrow, cardiac and skeletal muscle, and eye. Its lead product candidate is HMI-102, which is in Phase 2 pheNIX clinical trial, a gene therapy for the treatment of phenylketonuria (PKU) in adults. The company also develops HMI-103 for the treatment of PKU in pediatric patients; HMI-202 to treat metachromatic leukodystrophy; and HMI-203 for the treatment of mucopolysaccharidosis type II, as well as HMI-104 for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Homology Medicines, Inc. was incorporated in 2015 and is headquartered in Bedford, Massachusetts.」