Founded in 1996,the American Society of Gene&Cell Therapy is the world's largest non-profit medical organization dedicated to the research of gene and cell therapy.It is committed to advancing the science,technology,and clinical application of gene and cell therapy and is the most authoritative and credible institution in the global field of gene and cell therapy.The ASGCT Annual Meeting,as the premier event in the global field of gene and cell therapy,attracts thousands of scientists,clinicians,and industry leaders from around the world.It explores the cutting-edge progress and future development directions of gene therapy,promotes exchanges and cooperation among academia,industry,and regulatory authorities,and drives innovation and development in the global field of gene and cell therapy.
Oral Presentation：
Safety and Efficacy of AAV9-Mediated Gene Therapy GC301 in ERT-Stabilized Infantile-Onset Pompe Disease: An Open-Label, Single-Arm Study with Over 12 Months of Follow-Up
Title: Gene Therapy Trials - In-Vivo Gene Therapy Modification
Date: May 16, 2025
Time: 3:45 PM - 5:40 PM(Central Time)
Room: Room 393-396
Genecradle Therapeutics' AAV9 Vector Drug GC301 Injection for Infantile Pompe Disease Will Release Over 12 Months of Clinical Follow-Up Data at the Conference. The research indicates that a single intravenous injection of GC301 can reduce enzyme usage, significantly enhance patients' muscle strength and cardiac function, and maintain good safety. This achievement marks China's leap from "following" to "leading" in the field of lysosomal storage disease gene therapy, bringing hope for a "one-time cure" to the global Pompe disease community.
Poster Presentation：
1. Innovative Intrathecal AAV9-mediated Gene Therapy for Spinal Muscular Atrophy: Phase I/II Safety and Efficacy Topline Results
AMA191
Title: Wednesday Poster Reception
Date: May 14, 2025
Time: 5:30 PM - 7:00 PM(Central Time)
Room: Poster Hall Hall I2
Major First Release: Genecradle Therapeutics will publicly disclose for the first time at an international conference its independently developed intrathecal AAV9 gene therapy for Type 2 spinal muscular atrophy (SMA) in Phase I/II clinical trial topline data. The study employs a novel AAV9 vector, optimizing the administration route to reduce therapeutic dosage and enhance central nervous system expression efficiency, preliminarily verifying its significant efficacy and safety in SMA patients. As the first SMA gene therapy in China to use intrathecal injection, this innovative design offers patients a better treatment option.
2. Dosage Effects on Circulating GAA as a Pharmacodynamic Biomarker for systemically delivered rAAV9-coGAA Gene Therapy in Pompe Disease
AMA289
Title: Tuesday Poster Reception
Date: May 13, 2025
Time: 6:00 PM - 7:30 PM(Central Time)
Room: Poster Hall Hall I2
Precision Therapy: A New Stage in Predicting Pompe Disease Efficacy. A systematic evaluation of the dynamic relationship between rAAV9-coGAA dosing and circulating GAA enzyme levels provides crucial data support for efficacy monitoring and individualized dosing schemes in Pompe disease gene therapy.
3. A Novel Mmachc c.80A>G Mouse Model: Insights of Mitochondrial Dysfunction in Skeletal Muscle and Potential AAV-mediated gene therapy for cblC
AMA312
Title: Thursday Poster Reception
Date: May 15, 2025
Time: 5:30 PM - 7:00 PM (Central Time)
Room: Poster Hall Hall I2
Mechanism Exploration: Breakthrough in the cblC Disease Model. Mechanistic studies based on the Mmachc c.80A>G mutation mouse model reveal the central role of mitochondrial dysfunction in cblC methylmalonic acidemia and validate the potential efficacy of AAV gene therapy. This research offers key experimental evidence for the mechanism elucidation and drug development of inherited metabolic diseases.
*The selected abstracts can be viewed on the ASGCT website(https://annualmeeting.asgct.org/abstracts).
ASGCT 2025 is not only an important platform for Genecradle Therapeutics to showcase its scientific research breakthroughs to the world but also an opportunity for Chinese gene therapy to integrate into the global innovation ecosystem.Looking ahead,the company will continue to adhere to its core philosophy of"Originating from Needs,Benefiting Patients,"promoting cutting-edge therapies across borders to benefit more patients and injecting innovative momentum from Chinese technology into the global community of health.