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LY-M001

Phase 1

Gaucher Disease Type 1 | Gene therapy | Metabolic |Co-Diagnostics, Inc.|Last Updated: Jan 26, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDBiomarker
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06818838A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher DiseasePHASE1 RECRUITING 12 — — Jul 5, 2024Jul 30, 2031Jan 26, 20263 China
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Study Endpoints
Primary Endpoints
Phase I: Incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks after LY-M001 infusion
From enrollment to 52 weeks after administration

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.

Phase I: Incidence rate of dose-limiting toxicity (DLT) events determined by the data safety review committee (SRC) within at least 28 days after LY-M001 infusion.
From enrollment to 52 weeks after administration

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.The adverse events defined as dose-limiting toxicity (DLT) have been clearly specified in the protocol.

Phase I: Liver function levels (including alanine aminotransferase [ALT], aspartate aminotransferase [AST], total bilirubin [TBIL], alkaline phosphatase [ALP], gamma-glutamyl transferase [GGT]) within 52 weeks after LY-M001 infusion.
From enrollment to 52 weeks after administration

Incidence rate of liver function-related adverse events evaluated by CTCAE V5.0.

Phase II: Blood glucocerebrosidase (GCase) activity level.
From enrollment to 52 weeks after administration

Evaluation based on the detected values of blood glucocerebrosidase（GCase).

Phase II: The incidence rates of adverse events (AEs) and serious adverse events (SAEs), as well as the occurrence of abnormalities in 12-lead electrocardiogram (ECG) findings, vital signs, laboratory test parameters, and physical examination results.
From enrollment to 52 weeks after administration

Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.

Secondary Endpoints
Phase I: Liver volume and spleen volume (if applicable)
From enrollment to 52 weeks after administration
Phase I: Hemoglobin levels
From enrollment to 52 weeks after administration
Phase I:Bone mineral density (BMD) after administration
From enrollment to 52 weeks after administration
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Phase I: LY-M001 BackdoseEXPERIMENTALParticipants receive a single, peripheral intravenous (IV) infusion of LY-M001 at backdose.
Phase I: LY-M001 Dose group 1EXPERIMENTALParticipants receive a single, peripheral intravenous (IV) infusion of LY-M001 at dose group 1.
Phase I: LY-M001 Dose group 2EXPERIMENTALParticipants receive a single, peripheral intravenous (IV) infusion of LY-M001 at dose group 2.
Phase II: LY-M001 at the recommended doseEXPERIMENTALParticipants receive a single, peripheral IV infusion of LY-M001 at the recommended dose.
Interventions
NameTypeDescription
LY-M001GENETICSingle Intravenous Infusion of LY-M001 Injection.
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Eligibility Criteria
Age Range18 Years — 60 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: 1. Age ≥ 18 years and ≤ 60 years, male or female. 2. 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. 3. Patients with confirmed double mutations in the...

Countries:China
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