Lingyi Biotech Showcases Groundbreaking Clinical Advancements in Next-Generation Regulatable Gene Therapies at ASGCT 2025

Philadelphia, USA (May 13-17, 2025, EDT) - Lingyi Biotech Co., Ltd. ("Lingyi Biotech") showcased its latest research achievements at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT 2025), engaging in profound academic exchanges with thousands of global industry leaders, researchers, and clinical experts. As the world's premier platform for gene and cell therapy, the ASGCT annual meeting witnessed Lingyi Biotech's R&D capabilities in breaking through clinical bottlenecks with original technologies, with its differentiated pipeline layout and global innovation pathway garnering widespread attention.

 

 

The company delivered oral presentations in the Late-Breaking Abstracts session, reporting clinical trial results for:

  1. LY-M003: Investigational gene therapy for Wilson disease (hepatolenticular degeneration)

  2. LY-M001: Investigational gene therapy for Gaucher disease type 1

Both programs demonstrated substantial scientific merit and clinical significance.

 

 

LY-M003: Next-Generation Regulatable Gene Therapy Redefining Treatment Standards for Wilson's Disease

As the world's first AAV gene therapy employing copper-ion responsive dynamic expression technology, LY-M003 utilizes the proprietary CREATE™ (Copper-Responsive Engineering of ATP7B Expression) system to achieve on-demand expression of therapeutic proteins at the fundamental mechanism level, enabling dynamic coordination between therapeutic efficacy and pathological severity in Wilson's disease patients, thereby better ensuring long-term safety and effectiveness of the gene therapy.

• Clinical Highlights

  • All 7 enrolled patients demonstrated excellent infusion tolerance

  • No serious adverse events (SAEs) or anti-ATP7B antibodies (ADA) reported

  • Majority of patients completely discontinued standard medication

  • Patients with neurological symptoms showed significant improvement in UWDRS scores (writing ability, speech function, etc.)

  • Serum ceruloplasmin levels increased markedly

  • Urinary copper levels normalized in some patients

  • Represents potential transition from chronic pharmacotherapy to durable therapeutic effect

 

LY-M001: World-leading Clinical Advances in Gaucher Disease Treatment
For Gaucher disease (GD), LY-M001 utilizes an AAV vector to express GCaseLY, a glucocerebrosidase with higher protein stability and lower immunogenicity, combined with proprietary gene expression technology to fundamentally address issues in conventional enzyme replacement therapy (ERT) such as frequent injections and insufficient improvement of certain organ symptoms. 

• Clinical Highlights

  • Completed enrollment of 11 patients with Gaucher disease type 1 (most advanced clinical program globally)

  • Favorable safety profile: No treatment-related SAEs or anti-GCaseLY antibody development

  • Peripheral blood GCase activity returned to normal levels within 1 week after infusion

  • Progressive reduction in disease biomarker glucosylsphingosine (Lyso-GL1)

  • Clinically meaningful improvements:

    • Increased hemoglobin concentrations and platelet counts

    • Reduced hepatosplenomegaly (liver and spleen volumes)

    • Alleviation of disease-related symptoms (abdominal distension, fatigue)

  • All patients resumed normal work or study

  • Demonstrates potential to modify disease progression

 

Perspective
Dr. Qing Lin, Founder and CEO of Lingyi Biotech, stated: "Our ASGCT presentations mark a milestone in Lingyi’s 'Source Innovation + Global Synchronization' strategy. Both therapies overcome traditional limitations in gene expression control while demonstrating transformative 'single-dose, lifelong benefit' potential—aligning with cutting-edge directions in global gene therapy development. With FDA orphan drug and pediatric rare disease designations for LY-M001 and LY-M003, we are accelerating pipeline advancement to build a precision medicine ecosystem for monogenic disorders."