Gene therapy injection aimed at slowing ALS enters human testing
NCT ID NCT07290062
First seen Jun 25, 2026 · Last updated Jul 07, 2026 · Updated 4 times
Summary
This early-stage trial is testing a single injection of a gene therapy called INS1202, given into the spinal fluid, for people with ALS (Lou Gehrig's disease). The study includes 23 adults, some with a specific genetic mutation (SOD1) and some without known genetic causes. The main goal is to check safety and find the right dose, not yet to prove the treatment works.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- INS1202 (gene therapy given as a single injection into the spinal fluid)
- What this could lead to
- If safe and effective, this could point toward a treatment that slows or stops the progression of ALS, a devastating nerve disease.
- What could go wrong
- This is a very early Phase 1 trial with only 23 people, so it is primarily checking safety, not whether it works. Gene therapies can have unexpected side effects, and the benefit may be small or absent.
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.
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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USA001
RECRUITINGColumbia, Missouri, 65211, United States
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USA002
RECRUITINGPalo Alto, California, 94304, United States
Contact Email: •••••@•••••
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USA004
RECRUITINGLa Jolla, California, 92037, United States
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USA006
RECRUITINGPhiladelphia, Pennsylvania, 19107, United States
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USA007
RECRUITINGColumbus, Ohio, 43221, United States
Contact Email: •••••@•••••
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