AccurEdit Therapeutics to Present Groundbreaking Research at ASGCT 2025
AccurEdit Therapeutics, an emerging leader in the field of gene editing, is poised to make significant contributions to the upcoming 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) scheduled to take place from May 13 to May 17, 2025, in New Orleans, Louisiana. The company announced that three of its groundbreaking abstracts have been selected for presentation, including one oral session dedicated to discussing its flagship product, ART001.
ART001 is redefining the landscape of in vivo gene editing. Recognized as a pioneering lipid nanoparticle (LNP)-based therapy, ART001 is the only product of its kind that has successfully cleared Investigational New Drug (IND) requirements in both the United States and China. Presently, ART001 is advancing through phase I and phase IIa clinical trials in China while simultaneously entering phase I studies in the U.S.
During the oral presentation scheduled for Friday, May 16, 2025, from 5:15 PM to 5:30 PM CT, attendees will gain insights into the development and interim clinical outcomes of ART001, specifically its application for hereditary ATTR (transthyretin amyloidosis). This session is expected to illuminate the promising efficacy of the therapy, engaging the audience with real-world data and clinical findings that underline the potential of ART001 as a transformative treatment option.
In addition to the oral presentation, AccurEdit Therapeutics has two noteworthy poster presentations lined up. The first poster will focus on ART002, a single-dose gene editing therapy that has shown remarkable results in patients battling severe heterozygous familial hypercholesterolemia (HeFH). The data presented indicates that ART002 has achieved an average reduction of nearly 90% in PCSK9 levels and a 56% reduction in LDL-C levels among patients with critically high cholesterol baselines, above 6 mM. Presenting on this topic during the poster session on May 13, 2025, from 6:00 PM to 7:30 PM CT, AccurEdit aims to showcase the safety and efficacy of ART002, positioning it as a potential breakthrough in cholesterol management and cardiovascular health.
The second poster presentation will highlight advancements in the ARTbase-A1™ platform, which is uniquely owned by AccurEdit and has recently been granted patents in both China and the U.S. This novel base editor technology represents a significant leap forward in the development of non-viral gene editing approaches, reinforcing AccurEdit's commitment to innovation in therapeutic solutions.
Expressing enthusiasm about this opportunity, Yongzhong Wang, Ph.D., CEO of AccurEdit Therapeutics, stated, "We thank the organizing committee of ASGCT for the opportunity to present our latest data on our pipeline products, which have best-in-class potential and novel gene-editing tools with superior performance. We look forward to engaging with peers and potential collaborators to further discuss the opportunities to provide efficient, safe, and affordable in vivo gene therapeutics for our patients across the globe."
Founded in 2021, AccurEdit Therapeutics is dedicated to advancing in vivo gene editing technologies through innovative delivery systems, notably LNPs. The company emphasizes the importance of providing transformative treatment options that are accessible and cost-effective.
AccurEdit's robust clinical pipeline is focusing on genetic rare diseases and common disorders that presently lack effective treatment options. Its groundbreaking work culminated in August 2023 when ART001 became the first LNP-delivered in vivo gene editing medication to enter human clinical trails in China. The excitement continued as, by August 2024, ART001 earned U.S. FDA approval, representing a significant milestone in its journey. February 2025 marked another achievement for AccurEdit when ART002 emerged as the world's first in vivo gene editing therapy demonstrating therapeutic saturation in human subjects, achieving substantial LDL-C reductions in patients with extremely high cholesterol levels.
With ART001 and ART002 presenting promising safety and efficacy data, AccurEdit Therapeutics is on the verge of establishing its therapies as global benchmarks in gene editing technology, sincerely aiming to change the landscape of treatment for patients worldwide.
ART001 is redefining the landscape of in vivo gene editing. Recognized as a pioneering lipid nanoparticle (LNP)-based therapy, ART001 is the only product of its kind that has successfully cleared Investigational New Drug (IND) requirements in both the United States and China. Presently, ART001 is advancing through phase I and phase IIa clinical trials in China while simultaneously entering phase I studies in the U.S.
During the oral presentation scheduled for Friday, May 16, 2025, from 5:15 PM to 5:30 PM CT, attendees will gain insights into the development and interim clinical outcomes of ART001, specifically its application for hereditary ATTR (transthyretin amyloidosis). This session is expected to illuminate the promising efficacy of the therapy, engaging the audience with real-world data and clinical findings that underline the potential of ART001 as a transformative treatment option.
In addition to the oral presentation, AccurEdit Therapeutics has two noteworthy poster presentations lined up. The first poster will focus on ART002, a single-dose gene editing therapy that has shown remarkable results in patients battling severe heterozygous familial hypercholesterolemia (HeFH). The data presented indicates that ART002 has achieved an average reduction of nearly 90% in PCSK9 levels and a 56% reduction in LDL-C levels among patients with critically high cholesterol baselines, above 6 mM. Presenting on this topic during the poster session on May 13, 2025, from 6:00 PM to 7:30 PM CT, AccurEdit aims to showcase the safety and efficacy of ART002, positioning it as a potential breakthrough in cholesterol management and cardiovascular health.
The second poster presentation will highlight advancements in the ARTbase-A1™ platform, which is uniquely owned by AccurEdit and has recently been granted patents in both China and the U.S. This novel base editor technology represents a significant leap forward in the development of non-viral gene editing approaches, reinforcing AccurEdit's commitment to innovation in therapeutic solutions.
Expressing enthusiasm about this opportunity, Yongzhong Wang, Ph.D., CEO of AccurEdit Therapeutics, stated, "We thank the organizing committee of ASGCT for the opportunity to present our latest data on our pipeline products, which have best-in-class potential and novel gene-editing tools with superior performance. We look forward to engaging with peers and potential collaborators to further discuss the opportunities to provide efficient, safe, and affordable in vivo gene therapeutics for our patients across the globe."
Founded in 2021, AccurEdit Therapeutics is dedicated to advancing in vivo gene editing technologies through innovative delivery systems, notably LNPs. The company emphasizes the importance of providing transformative treatment options that are accessible and cost-effective.
AccurEdit's robust clinical pipeline is focusing on genetic rare diseases and common disorders that presently lack effective treatment options. Its groundbreaking work culminated in August 2023 when ART001 became the first LNP-delivered in vivo gene editing medication to enter human clinical trails in China. The excitement continued as, by August 2024, ART001 earned U.S. FDA approval, representing a significant milestone in its journey. February 2025 marked another achievement for AccurEdit when ART002 emerged as the world's first in vivo gene editing therapy demonstrating therapeutic saturation in human subjects, achieving substantial LDL-C reductions in patients with extremely high cholesterol levels.
With ART001 and ART002 presenting promising safety and efficacy data, AccurEdit Therapeutics is on the verge of establishing its therapies as global benchmarks in gene editing technology, sincerely aiming to change the landscape of treatment for patients worldwide.
Topics Health）
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