Accuredit Therapeutics US limited
Monday, June 03, 2024
Company Presentation
Cell and Gene Therapy and Genome Editing
Company Presentation Theater 1
AccurEdit focuses on the development and commercialization of safe, effective and targeted in vivo gene editing and delivery technologies for hereditary and acquired diseases that are severe or life-threatening to patients worldwide.
AccurEdit has established an industry-leading end-to-end technology platform for in vivo gene editing therapy with focuses on RNA synthesis, editing, and delivery technologies.
In October 2023, AccurEdit became the first and only company in China to demonstrate safe and effective systemic CRISPR gene editing in humans.
AccurEdit follows a patient-oriented philosophy in product development. At AccurEdit we believe in a commercial model of sustainable growth through rapid development of single-dose curative therapy for applicable diseases via a highly efficient technology platform, rather than through patient's repeated or even lifelong medication.
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Company Website:
https://www.accuredit.com/
Lead Product in Development:
ART001
ART001 is targeting ATTR amyloidosis, which arises from the deposition of fibrils formed by abnormal TTR protein. ATTR amyloidosis is a rare, progressive and fatal disease, which occurs when mutation in the TTR gene causes the liver to produce structurally unstable transthyretin proteins with a propensity to misfold, resulting in the formation of amyloid fibrils that deposit in various organs and peripheral nerves and cause them to function abnormally. Current treatment options include small molecule-based stabilizers, ASOs and RNAi, which requires repeated medication. ART001, an in vivo gene editing product delivered by LNP, provides a single dose cure for life-long benefits. According to the most recent IIT data, ATTR patients treated with a single dose of ART001 achieved over 90% reduction in serum TTR level after 4 weeks. ART001 is well-tolerated and demonstrated an overall outstanding safety profile. No infusion-related reaction was observed in any of the patients treated. No >Grade 1 liver enzyme elevation was observed. In studies using primary human hepatocytes, no off-target editing was observed in ART001 even at up to ~80 times of EC90.
Company HQ City
Cambridge
Company HQ State
MA
Company HQ Country
United States
CEO/Top Company Official
Yongzhong Wang
Development Phase of Primary Product
Phase I
Speakers
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