Gene therapy SPOT-03 enters human testing for duchenne muscular dystrophy

NCT ID NCT07188012

Recruiting now Disease control Sponsor: Shanghai Siponuoyin Biotechnology Co Ltd Source: ClinicalTrials.gov ↗

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

Summary

This early-phase trial tests a gene therapy called SPOT-03 in 9 boys with Duchenne muscular dystrophy (DMD), aged 2 to 8. The main goal is to see if the treatment is safe and tolerable. Researchers will also check if it increases dystrophin protein levels in muscles, which could help slow the disease.

What this could mean

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

Active substance: SPOT-03 (gene therapy)
What this could lead to: If successful, this could point toward a treatment that helps boys with Duchenne muscular dystrophy produce dystrophin protein, potentially slowing muscle damage.
What could go wrong: This is a very early, small trial (9 participants) focused on safety, not effectiveness. Gene therapies can have side effects, and it may not work as hoped.

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.

Duchenne muscular dystrophy

As listed by the trial registrant

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

DUCHENNE MUSCULAR DYSTROPHY (DMD)

Contacts and locations

Locations

Shanghai Children's Medical Center
RECRUITING

Shanghai, Shanghai Municipality, China

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