Gene therapy for SMA tested in real-world settings of poorer nations

NCT ID NCT07265232

Recruiting now Disease control Sponsor: Lantu Biopharma Source: ClinicalTrials.gov ↗

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

Summary

This study is testing a gene therapy called vesemnogene lantuparvovec for spinal muscular atrophy (SMA) in low- and middle-income countries. Researchers will track safety and whether children reach motor milestones like sitting or walking. The trial involves 15 participants and aims to see if the treatment works outside of wealthy countries.

What this could mean

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

Active substance: vesemnogene lantuparvovec (a gene therapy)
What this could lead to: If successful, this could show that gene therapy for SMA is safe and effective in low-resource settings, potentially expanding access to treatment.
What could go wrong: This is a small, early real-world study with only 15 participants, so results may not apply broadly. Gene therapy carries risks like immune reactions and unknown long-term effects.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

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

SPINAL MUSCULAR ATROPHY (SMA)

Contacts and locations

Locations

Tzu chi hospital
RECRUITING

Jakarta, Indonesia, 14470, Indonesia

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