Gene therapy trial aims to fix enzyme defect in gaucher disease

NCT ID NCT05324943

Completed Disease control Sponsor: Spur Therapeutics Source: ClinicalTrials.gov ↗

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

Summary

This early-stage trial tested a new gene therapy called FLT201 in 10 adults with Gaucher disease type 1. The therapy uses a harmless virus to deliver a working copy of the gene that produces a missing enzyme. The main goal was to check safety and see if the treatment can boost enzyme levels and reduce disease buildup.

What this could mean

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

Active substance: FLT201 gene therapy
What this could lead to: If successful, this could lead to a one-time gene therapy that helps the body produce the missing enzyme, potentially reducing the need for regular infusions.
What could go wrong: This is a very early, first-in-human trial with only 10 participants. It primarily tests safety, not effectiveness. Gene therapies can have unexpected side effects, and long-term benefits are uncertain.

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.

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

Hospital Quironsalud Zaragoza

Zaragoza, Spain
Hospital de Clinicas de Porto Alegre (HCPA)

Porto Alegre, Brazil
Kaiser Permanente

Los Angeles, California, 90027, United States
Lysosomal Rare Disorders Research and Treatment Center

Fairfax, Virginia, 22030-6066, United States
Rabin Medical Center - PPDS

Petah Tikva, Israel
Royal Free Hospital

London, United Kingdom
Salford Royal Hospital

Salford, United Kingdom
Shaare Zedek Medical Center

Jerusalem, Israel
SphinCS

Höchheim, Germany
Tel Aviv Sourasky Medical Center

Tel Aviv, Israel

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