Gene therapy injection aims to halt fatal brain disease in babies
NCT ID NCT04411654
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase trial is testing a single-dose gene therapy called LY3884961 in 7 infants with Type 2 Gaucher disease, a severe and rapidly fatal genetic disorder. The therapy is injected into the fluid around the brain and spinal cord to deliver a working copy of the faulty gene. Infants also receive immune-suppressing drugs to prevent rejection. The study will monitor safety, side effects, and whether the treatment can slow disease progression over 5 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- LY3884961 (a gene therapy given as a single injection into the fluid around the brain and spinal cord)
- What this could lead to
- If successful, this could point toward a treatment that slows or stops the severe brain damage caused by Type 2 Gaucher disease, potentially allowing infants to live longer and with fewer symptoms.
- What could go wrong
- This is a very early, small trial with only 7 infants, so results may not apply to others. The gene therapy is given directly into the central nervous system, which carries risks like inflammation or immune reactions. Lifelong immunosuppressant drugs are needed alongside, so it is not a cure.
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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
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Locations
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Children's Hospital of Pittsburgh, 4401 Penn Avenue
Pittsburgh, Pennsylvania, 15224, United States
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Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030, United States
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Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road
Manchester, M13 9WL, United Kingdom
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UCSF Benioff Children's Hospital, 747 52nd St
Oakland, California, 94609, United States
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University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue
Minneapolis, Minnesota, 55454, United States
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