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.
Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.