Editas Medicine Inc., a pioneering gene editing company, has announced new _in vivo_ data showcasing the potential of their gene upregulation strategy in hematopoietic stem cells (HSCs). The data, presented at the American Society of Gene and Cell Therapy Annual Meeting, revealed therapeutically relevant levels of _HBG1/2_ promoter editing achieved with a single dose of proprietary targeted lipid nanoparticles in humanized mice and non-human primates. This clinically validated approach aims to upregulate fetal hemoglobin (HbF) and is in pre-clinical development as a novel treatment for sickle cell disease and beta thalassemia. The results highlight the potential of Editas' strategy to address significant unmet medical needs in these diseases.
免责声明：投资有风险，本文并非投资建议，以上内容不应被视为任何金融产品的购买或出售要约、建议或邀请，作者或其他用户的任何相关讨论、评论或帖子也不应被视为此类内容。本文仅供一般参考，不考虑您的个人投资目标、财务状况或需求。TTM对信息的准确性和完整性不承担任何责任或保证，投资者应自行研究并在投资前寻求专业建议。