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China-based AccurEdit Therapeutics (Suzhou) Co., Ltd announced receiving Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its investigational drug ART001, targeting transthyretin amyloidosis (ATTR). This marks a significant milestone in the development of this innovative gene-editing therapy. Drug Profile and MechanismART001 is the first non-viral…
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Suzhou-based gene editing specialist AccurEdit Therapeutics announced that its in vivo gene editing product, ART002, has achieved drug saturation and safely reduced low-density lipoprotein cholesterol (LDL-C) with a single dose in a multi-center, open, single-arm, exploratory investigator-initiated trial (ITT) for heterozygous familial hypercholesterolemia. This marks the first time an in…
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Germany-based Bayer AG (ETR: BAYN) has announced that Chinese companies Epigenic Therapeutics, Shanghai Immunocan Biotech Co., Ltd., and AccurEdit Therapeutics will be housed at its Bayer Co.Lab Shanghai. This move aims to leverage Bayer’s global innovation and cooperation network, connecting global pharmaceutical industry resources to boost cell and gene therapy…
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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…
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SUZHOU—AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading Chinese specialist in gene editing therapies, has announced that it has received approval from the US Food and Drug Administration (FDA) for its investigational drug ART001, which targets transthyretin amyloidosis. This milestone marks ART001 as the first in vivo gene-edited drug based on…
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AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading specialist in gene editing therapies based in China, has received approval from the Center for Drug Evaluation (CDE) for its investigational drug ART001. This drug is designed to target transthyroxine protein amyloidosis, a severe genetic disorder. ART001 marks a significant advancement in China’s…
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AccurEdit Therapeutics (Suzhou) Co., Ltd., a leading gene editing therapy specialist based in China, has published the efficacy and safety data for its drug candidate ART001, which is being investigated as a treatment for transthyroxine protein amyloidosis. ART001, the first non-viral vector in vivo gene-edited drug in China to enter…
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China-based gene therapy start-up Accuredit Therapeutics (Suzhou) Co., Ltd has announced the first dosing of its investigational drug ART001 in seven subjects with transthyretin amyloidosis (ATTR), as part of an investigator-initiated trial (ITT). The drug candidate has demonstrated good safety and achieved a clinically significant decrease in transthyroxine (TTR) protein…