{"id":16457,"date":"2024-12-03T18:51:31","date_gmt":"2024-12-03T10:51:31","guid":{"rendered":"https:\/\/flcube.com\/?p=16457"},"modified":"2024-12-03T18:51:38","modified_gmt":"2024-12-03T10:51:38","slug":"accuredit-therapeutics-art001-shows-over-90-ttr-reduction-in-attr-amyloidosis-trial","status":"publish","type":"post","link":"https:\/\/flcube.com\/?p=16457","title":{"rendered":"AccurEdit Therapeutics’ ART001 Shows Over 90% TTR Reduction in ATTR Amyloidosis Trial"},"content":{"rendered":"\n

Suzhou-based gene editing specialist AccurEdit Therapeutics has announced positive results from an ongoing investigator-initiated trial (ITT) for its in vivo gene editing product, ART001. This lipid nanoparticle (LNP) delivered treatment is designed for transthyretin amyloidosis (ATTR), a rare, progressive, and fatal disease. In subjects who completed at least 48 weeks of follow-up, ART001 demonstrated a good safety profile and an over 90% reduction in serum TTR levels.<\/p>\n\n\n\n

Understanding ATTR Amyloidosis and the Impact of ART001<\/strong>
ATTR amyloidosis occurs due to mutations in the TTR gene, leading to the production of unstable transthyretin proteins that are prone to misfolding. This results in the formation of amyloid fibrils that deposit in various organs and peripheral nerves, causing abnormal function. The positive outcomes of the trial indicate that ART001 could be a significant advancement in the treatment of this disease.<\/p>\n\n\n\n

ART001’s Safety and Efficacy Profile<\/strong>
No infusion-related reactions were observed in subjects treated with ART001. Additionally, in studies using primary human hepatocytes, ART001 showed no off-target editing even at concentrations up to approximately 80 times the EC90. This highlights the precision and safety of the gene editing therapy.<\/p>\n\n\n\n

Global Clinical Trial Clearance for ART001<\/strong>
ART001 received clearance for clinical trials in China in July and the United States in August of this year, making it the only gene editing therapy of its kind with clinical approval in both countries. This milestone underscores the potential global impact of AccurEdit’s therapy in addressing ATTR amyloidosis.-Fineline Info & Tech<\/a><\/p>\n\n\n\n

<\/p>\n","protected":false},"excerpt":{"rendered":"

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