One-Time gene therapy could change gaucher treatment forever

NCT ID NCT06818838

Recruiting now Disease control Sponsor: Lingyi Biotech Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new gene therapy called LY-M001 for adults with Gaucher disease type 1, a genetic disorder that causes organ damage and other health problems. The therapy uses a harmless virus to deliver a working copy of the GBA1 gene to liver cells, aiming to restore the missing enzyme. Twelve participants will receive a single intravenous dose, and researchers will monitor safety and effectiveness over a year.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: LY-M001 (a gene therapy using a modified virus to deliver a working GBA1 gene to liver cells)
What this could lead to: If successful, this could provide a long-lasting treatment that reduces or eliminates the need for regular enzyme replacement therapy for Gaucher disease type 1.
What could go wrong: This is an early-phase trial with only 12 participants, so results may not apply to everyone. Gene therapies can cause immune reactions or liver problems, and long-term effects are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

anemia Gaucher disease Gaucher disease type I

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GAUCHER DISEASE TYPE 1

Contacts and locations

Locations

Guangzhou First People's Hospital
RECRUITING

Guangzhou, Guangdong, 510000, China
Hematology Hospital, Chinese Academy of Medical Sciences
RECRUITING

Tianjin, Tianjin Municipality, 300011, China
Shanxi Bethune Hospital
RECRUITING

Taiyuan, Shanxi, 030000, China

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