Drug Profile

LY M001

Alternative Names: LY-M001

Latest Information Update: 17 Feb 2026

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At a glance

Originator Lingyi Biotech
Class Gene therapies
Mechanism of Action GBA protein replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

Highest Development Phases

Phase I/II Gaucher's disease type I

Most Recent Events

06 Dec 2025 Efficacy and adverse events data from a clinical trial in Gaucher's disease type I presented at the 67^thAmerican Society of Hematology Annual Meeting and Exposition (ASH-2025)
01 Aug 2024 Phase-0 for Gaucher's disease type I (In children, In adolescents) in China (IV) (NCT06528080)
30 Oct 2023 Clinical trials in Gaucher's disease type I (In adults) in China (IV) (NCT06162338)

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