LY-M001 / Lingyi Biotech - LARVOL DELTA

First-in-Pediatric Clinical Evaluation of AAV8-GBA1 Gene Therapy (LY-M001) for Type 1 Gaucher Disease (ASGCT 2026) - P1 | "No occurrence of anti-GCase antibodies supports low immunogenicity and the potential for durable efficacy. Collectively, these findings support further clinical evaluation of LY-M001 as a potential disease-modifying therapy for pediatric Gaucher disease."

Clinical • Gene therapy • Gaucher Disease • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Pediatrics • Rare Diseases • Type 1 Gaucher Disease • GBA1

A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease (clinicaltrials.gov) - P1 | N=9 | Active, not recruiting | Sponsor: Shanghai Jiao Tong University School of Medicine | Recruiting ➔ Active, not recruiting | Trial completion date: Aug 2029 ➔ May 2030 | Trial primary completion date: Aug 2025 ➔ May 2027

Enrollment closed • Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Pediatrics • Type 1 Gaucher Disease • GBA1

GD: A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Lingyi Biotech Co., Ltd. | N=18 ➔ 12

Enrollment change • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Safety and activity of gene therapy with AAV8 GBA1 (LY-M001)on type I adults gaucher disease (ASH 2025) - P=N/A | "After one week of prophylactic prednisone (1 mg/kg/day),participants received a single intravenous infusion of LY-M001 at either 5×10¹² vg/kg (Cohort 1, n=1) or1.5×10¹³ vg/kg (Cohort 2, n=2)...In Cohort 2, one patient had transient ALTelevations (peak 96 IU/L) and delayed mild elevation (ALT 73 IU/L), managed with oral tacrolimus...This pilot study suggests that LY-M001 is well tolerated up to 21 months post-infusion, with encouragingbiochemical and clinical responses observed in the medium-dose cohort. LY-M001 holds potential as apromising gene therapy alternative to current GD treatments, addressing key unmet clinical needs."

Clinical • Gene therapy • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease • GBA • GBA1

Evaluation of Clinical Safety and Efficacy of LY-M001: A Phase I/II Trial of AAV8-Mediated Gene Therapy for Gaucher Disease Type I (ESGCT 2025) - No abstract available

Clinical • Gene therapy • P1/2 data • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

Evaluation of Clinical Safety and Efficacy of LY-M001: A Phase I/II Trial of AAV8-Mediated Gene Therapy for Gaucher Disease Type I (ASGCT 2025) - P=N/A, P1 | "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."

Clinical • Gene therapy • P1/2 data • Fatigue • Gaucher Disease • Gene Therapies • Genetic Disorders • Immune Modulation • Immunology • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Type 1 Gaucher Disease • GBA1

Evaluation of Clinical Safety and Efficacy of LY-M001: A Phase I/II Trial of AAV8-Mediated Gene Therapy for Gaucher Disease Type I (ASGCT 2025) - P=N/A, P1, P1/2 | "LY-M001 represents a novel AAV8-based gene therapy approach for Gaucher disease. Preliminary clinical data across different dosage groups indicate a strong safety profile and compelling efficacy. Enhanced GCase expression facilitates substrate clearance, leading to meaningful clinical benefits, including hematologic normalization, organ volume reduction, and improved patient-reported outcomes."

Clinical • Gene therapy • Late-breaking abstract • P1/2 data • Fatigue • Gaucher Disease • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Type 1 Gaucher Disease • GBA1

A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients with Gaucher Disease Type I (clinicaltrials.gov) - P=N/A | N=3 | Recruiting | Sponsor: He Huang | Trial completion date: Oct 2028 ➔ Dec 2031 | Trial primary completion date: Aug 2024 ➔ Dec 2026

Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

GD: A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients with Type I Gaucher Disease (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Lingyi Biotech Co., Ltd.

New P1/2 trial • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease

A multicenter, open, single-arm, single-dose, dose-escalation, and expanded Phase I/II study evaluating the safety, tolerability, and efficacy of LY-M001 injection in adult patients with type I Gaucher disease (ChiCTR) - P1/2 | N=18 | Recruiting | Sponsor: Hematology Hospital, Chinese Academy of Medical Sciences; Hematology Hospital, Chinese Academy of Medical Sciences

New P1/2 trial • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease • GBA1

Clinical Study of LY-M001 Injection in the Treatment of Adolescents With Type I Gaucher Disease (clinicaltrials.gov) - P1 | N=9 | Recruiting | Sponsor: Shanghai Jiao Tong University School of Medicine

New P1 trial • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease