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SUZHOU, China, May 12, 2025 /PRNewswire/ — AccurEdit Therapeutics today announced that its three abstracts have been selected for presentation, including one oral presentation on ART001, at the upcoming American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting being held from May 13-17, 2025, in New Orleans, LA.
ART001 is the only LNP-based in vivo gene editing product that has cleared INDs in both the US and China. It is currently in phase I/IIa studies in China and phase I study in the US.
The two poster presentations focus on ART002 and ARTbase-A1™, respectively. ART002 is AccurEdit Therapeutics’ second pipeline product that has achieved nearly 90% reduction in PCSK9 and 56% reduction in LDL-C on average in HeFH patients with critically high LDL-C baselines (>6 mM). ARTbase-A1™ is AccurEdit Therapeutics’ solely-owned base editor platform that has recently been granted patents in China and the US.
“We thank the organizing committee of ASGCT for the opportunity to present the latest data on our pipeline products with best-in-class potentials and novel gene-editing tools with industry-leading performances.” said Yongzhong Wang, Ph.D., CEO of AccurEdit Therapeutics. “We look forward to meeting our peers and potential collaborators to discuss the exciting perspective of delivering more efficient, safe and affordable in vivo gene therapeutics for our patients globally.”
Oral Presentation Details
Abstract Title: ART001: Development and Interim Clinical Outcomes of a CRISPR-Based In Vivo Gene-Editing Therapy for Hereditary ATTR
Oral Presentation Date/Time: Friday, May 16, 2025 at 5:15 PM – 5:30 PM CT
Room: Room 393-396
Final Abstract Number: AMA434
Poster Presentation Details
Abstract Title: Efficacy and Safety of ART002, a Single-dose Gene Editing Therapy, in Patients with Severe Heterozygous Familial Hypercholesterolemia
Poster Session Date/Time: Tuesday, May 13, 2025 at 6:00 PM – 7:30 PM CT
Room: Poster Hall Hall I2
Final Abstract Number: AMA562
Abstract Title: Engineering TadA-Derived Base Editors with Enhanced Potency and Safety for non-viral In Vivo Gene Editing
Poster Session Date/Time: Thursday, May 15, 2025 at 5:30 PM – 7:00 PM CT
Room: Poster Hall Hall I2
Final Abstract Number: AMA559
About AccurEdit Therapeutics:
AccurEdit Therapeutics, founded in 2021, focuses on developing in vivo gene editing technologies and therapeutics based on LNP (lipid nanoparticle) and other non-viral delivery systems. The company is dedicated to providing innovative one-time treatment solutions with cost advantages for patients worldwide.
The company boasts a professional team with full-lifecycle experience in successful biologics development and has established the world’s first clinically validated, industry-grade, end-to-end in vivo gene editing platform. It has systematically built intellectual property portfolios for related technologies and products, including ARTbase-A1™, a novel base editor that has been granted a U.S. patent.
AccurEdit Therapeutics has developed a pipeline targeting genetic rare diseases and refractory common disorders. In August 2023, its ATTR-targeted therapy ART001 became China’s first LNP-delivered in vivo gene editing drug to enter human clinical trials. By August 2024, ART001 further achieved another milestone as the first and only product of its kind in China to receive U.S. FDA clinical trial approval. In February 2025, ART002, targeting HeFH (PCSK9), emerged as the world’s first in vivo gene editing therapy to demonstrate therapeutic saturation in humans, effectively reducing LDL-C in patients with extremely high baseline levels. Both ART001 and ART002 have shown safety and efficacy data with the potential to become global best-in-class therapies.
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