ASGCT 2025| 凌意生物携创新成果亮相国际

 

 

 

 

2025年5月8日,全球领先的单基因遗传病药物开发企业——凌意(杭州)生物科技有限公司(以下简称“凌意生物”)宣布将出席2025年5月13日至17日在美国路易斯安那州新奥尔良举办的第28届美国基因与细胞治疗学会(ASGCT)年会。此次大会,凌意生物将发表6项基因治疗研发最新进展,其中2项以口头报告形式分别发表针对戈谢病I型的基因治疗药物LY-M001注射液和针对威尔逊病的基因治疗药物LY-M003注射液的临床研究成果。这标志着凌意生物在基因治疗领域的创新能力和临床转化效率获得国际认可。

 

 

 

 

01

Oral Presentation

 

 

Late-Breaking Clinical Progress

 

 

 

Evaluation of Clinical Safety and Efficacy of LY-M001: A Phase I/II Trial of AAV8-Mediated Gene Therapy for Gaucher Disease Type I

Date:May 16, 2025  

▶ Time:10:30–10:45 AM CT

▶ Room 288-290

LY-M001 is a novel AAV8-based therapeutic approach for Gaucher disease currently under investigation. Preliminary clinical data from various dosage groups demonstrate a favorable safety profile and promising efficacy outcomes. Enhanced GCase expression facilitates substrate clearance, leading to improvements in both Gaucher disease symptoms and patients' quality of life.

 

Revolutionizing Wilson’s Disease Treatment: Clinical Safety and Efficacy of LY-M003, A Copper-Responsive AAV Gene Therapy Vector

Date:May 17, 2025  

▶ Time:8:45–9:00 AM CT

▶ Room 391-392

LY-M003 is a novel AAV8-based therapeutic approach for Wilson’s disease currently under clinical investigation. Preliminary clinical data from various dosage groups thus far indicated a favorable safety profile and therapeutic efficacy on both peripheral and neurological symptoms, which underscores LY-M003’s profound potential in revolutionizing the therapeutic landscape for Wilson disease, heralding a new era of treatment possibilities.

 

 

 

 

 

 

02

Poster

 

 

 

🔹A Novel and Reliable In Vitro Copper-Excretion Potency Assay with Engineered Huh-7-CuEP Cells for Evaluating Gene Therapy Candidates in Wilson Disease

Date:May 13,2025

Time:6:00-7:30 PM CT

 

🔹LY-M003, An AAV Gene Therapy Product with Copper Responsive Engineering of ATP7B Expression (CREATE) for Wilson Disease

Date:May 14,2025

Time:5:30-7:00 PM CT

 

🔹Preclinical Evaluation of LY-N001 in an innovative GBA1-Associated Parkinson’s Disease Rat Model

Date:May 14,2025

Time:5:30-7:00 PM CT

 

🔹Establishment of The Inducible AAV Production Cell Line 293RSS2.0

Date:May 15,2025

Time:5:30-7:00 PM CT

 

ASGCT 2025不仅是凌意生物向全球展示其科研突破与临床价值的关键舞台,更是中国基因治疗技术深度参与全球创新生态的重要契机。未来,公司将继续践行以科学突破回应未竟之需,以治愈方案重塑生命希望的初心,通过跨国协作与开放创新,推动前沿疗法突破地域与技术的边界,让“一次性治愈”的愿景惠及全球更多单基因遗传病患者。凌意生物将携手国际伙伴,以中国智慧与创新动能推动基因治疗领域迈向更高维度,为构建人类健康共同体贡献可持续的科技力量。