SHANGHAI, CHINA / ACCESS Newswire / July 22, 2025 / Lingyi Biotech, a clinical-stage biotechnology company specializing in gene therapies for rare diseases, announced key clinical milestones at the 2025 American Society of Gene & Cell Therapy (ASGCT) annual meeting in New Orleans. The company highlighted its lead investigational programs, LY-M001 for Gaucher disease and LY-M003 for Wilson disease, both built on its next-generation AAV2.0 platform.

First-in-Class Gene Therapy for Gaucher Disease

LY-M001 is a gene therapy candidate based on a recombinant adeno-associated virus serotype 8 (rAAV8). It delivers a codon-optimized GBA1 gene driven by a proprietary liver-specific promoter to express a stabilized form of the GCaseLY enzyme.

Ongoing clinical trials, including Phase I/II studies, a pilot study, and the world's first adolescent trial for Gaucher disease, are being conducted across top Chinese medical institutions, including the Hematology Hospital of the Chinese Academy of Medical Sciences and Shanghai Children's Medical Center. Across dosing groups, patients have demonstrated improved substrate clearance, normalized hematologic indicators, reduced spleen and liver volumes, and an enhanced quality of life.

LY-M001 was recognized as "Rare Disease Drug of the Year" at the 2024 China Biopharmaceutical Industry Innovation Awards.

Targeted Therapy for Wilson Disease Using the CREATE™ System

LY-M003 employs Lingyi's proprietary CREATE™ system (Copper-Responsive Engineering of ATP7B Expression), which dynamically regulates ATP7B expression according to physiological copper levels. This adaptive control helps restore natural copper metabolism and ceruloplasmin production, enhancing safety and efficacy.

Early clinical results from The First Affiliated Hospital, Zhejiang University School of Medicine, show improvements in copper regulation and neurological symptoms, supporting LY-M003's potential as a best-in-class treatment for Wilson disease.

Expert Perspectives on Clinical Progress

Clinical leaders involved in the LY-M001 and LY-M003 trials have offered valuable insights into the early safety and efficacy signals.

Prof. Chaohui Yu, Deputy Dean at The First Affiliated Hospital, Zhejiang University School of Medicine, and Academic Leader of Gastroenterology, commented:

"LY-M003 injection, a groundbreaking achievement in gene therapy, has demonstrated an excellent safety profile in preliminary clinical observations. All enrolled participants exhibited good infusion tolerance with no serious adverse events (SAEs) reported. Although no significant changes were observed in serum ceruloplasmin expression levels, its enzymatic activity showed marked improvement, accompanied by a significant reduction in 24-hour urinary copper levels.

We look forward to obtaining more comprehensive safety and efficacy data on LY-M003 injection, which may provide a breakthrough treatment option for patients with Wilson's disease (hepatolenticular degeneration). Our hospital will continue to strengthen strategic collaborations with industry partners to accelerate the clinical translation and application of gene therapy technologies. These efforts aim to enhance public awareness of rare diseases and bring new hope to patients in need."

Prof. Fengkui Zhang of the Chinese Academy of Medical Sciences Haematological Diseases Hospital shared:

"LY-M001 has shown encouraging results in clinical trials. Participants tolerated the therapy well, with no serious adverse events related to the investigational product. Within just one week of a single dose, glucocerebrosidase activity returned to normal, and the key biomarker Lyso-GL1 steadily improved, indicating significant potential for clinical benefit.

Moving forward, the hospital will continue to strengthen industry-academia-research collaboration, driving innovation and clinical application of gene diagnosis and treatment technologies, and building a precision medicine ecosystem for genetic disorders."

Expanding Into Parkinson's Disease

Lingyi Biotech is also developing LY-N001, an AAV9-based gene therapy targeting GBA1-associated Parkinson's disease. By constitutively expressing a codon-optimized GBA1 gene, LY-N001 aims to address lysosomal dysfunction linked to neurodegeneration. Promising preclinical results in rodent and non-human primate models suggest strong potential for clinical impact in this prevalent disorder.

Strength in Platform Technology

Lingyi's proprietary AAV2.0 platform integrates:

• LY-Capsid, an AI-assisted capsid engineering system for tissue-specific gene delivery

• CREATE™, a regulatable gene expression system for safer dosing

• LY-293RSS, a next-generation manufacturing process designed for scalability and efficiency

This platform supports scalable and efficient production while addressing key delivery and durability challenges in gene therapy. The company also operates an in-house disease modeling and biomarker development platform, which accelerates rare disease drug discovery and validation.

Leadership and Global Strategy

Founded in 2021, Lingyi Biotech is led by a seasoned executive team with global biopharma experience and deep expertise in translational medicine. Key leaders include Founder and CEO Dr. Andrew Lin, Chief Scientific Officer Yixiong Chen, PhD, and Chief Financial Officer Chaoyi Zheng. Together, they bring decades of experience spanning research, development, and strategy in leading biotech organizations.

Since its founding, the company has advanced rapidly, supported by dual regulatory pathways in the U.S. (FDA) and China (NMPA). Its programs are built on validated preclinical models, deep disease insight, and a strategic focus on scalable manufacturing.

A development partnership with Zhejiang Zuoli Pharmaceutical Company further strengthens Lingy Biotech's capacity for clinical advancement and future commercialization.

About Lingyi Biotech

Lingyi Biotech is a clinical-stage biotechnology company that develops gene therapies for rare monogenic diseases using its proprietary AAV2.0 platform. Founded in 2021 and headquartered in China, Lingyi combines scientific innovation, global R&D experience, and a mission-driven approach to tackle some of the most urgent challenges in rare disease treatment. Its pipeline includes clinical-stage and preclinical programs in metabolic, neurologic, and ophthalmic disorders.

Media Contact:

Company: Lingyi Biotech Co., Ltd.

Website: www.lingyimed.com

Email: PR@lingyimed.com

Address: 160 Basheng Road, Building 7-4-2, Pudong New District, 200131 Shanghai, China

SOURCE: Lingyi Biotech Co., Ltd.