Gene therapy trial aims to fix enzyme defect in gaucher disease
NCT ID NCT05324943
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.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GAUCHER DISEASE TYPE 1 are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
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