This is a prospective single-center, open, single-arm, single-dose intravenous infusion study to evaluate the safety and initial efficacy, pharmacodynamic characteristics, immunogenicity, biodistribution, and viral shedding of LY-M001 injection.This study mainly includes the main study stage and the long-term follow-up study stage.
Age ≥ 18 years and ≤ 60 years, male or female.
The subjects should fully understand the purpose, nature and method of this study as well as possible adverse reactions, and sign the informed consent form (ICF) voluntarily.
Patients with GD1 who have confirmed double mutations in the Gba1 allele by laboratory testing and meet the standard for clinical diagnosis of Gaucher disease (i.e., reduced GCase enzyme activity to less than 30% of normal values).
The subjects were type I patients with Gaucher disease. Patients with type I Gaucher disease who had received specific treatment in the past required 5 half-lives of elution
AAV8 neutralizing antibody is strongly positive.
Patients with clinically suspected Gaucher disease type II (GD2) or Gaucher disease type III (GD3).
Active and progressive bone disease that is expected to require surgical treatment within the next 6 months.
Subject has idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), thrombocytopenia, anemia, hepatomegaly, splenomegaly, and/or osteoporosis unrelated to Gaucher disease as judged by the investigator.
Single group
LY-M001 Injection
Biological/VaccineWith 5.0 × 10\^12 vg/kg as the initial effective dose (first dose group), 1 to 2 subjects are expected to be included. The first dose group was enrolled by sentinel method, and the first subject in this group was observed for at least 28 days after receiving LY-M001 (dose-limited toxicity \[DLT\] observation period) to enroll the next subject. After the first participant completes the safety evaluation of dosing for at least 28 days, the investigator (PI) and the partner discuss the safety and efficacy data and decide to maintain the first dose or increase/reduce it to another dose group
Incidence of treatment- related adverse events
Number and severity of adverse events and serious adverse events and relationship to LY-M001
Incidence of events adjudicated as dose limiting toxicity (DLT)
Number and severity of dose limiting toxicity
Glucocerebrosidase (GCase) protein in plasma and GCase enzyme activity
Change from baseline in plasma GCase protein and GCase enzyme activity level
Glucosylsphingosine (Lyso-GL1)in plasma
Change from baseline in plasma Lyso-GL1 level
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