Gene therapy injection aimed at slowing ALS enters human testing

NCT ID NCT07290062

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 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.

Disclaimer Read more

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.

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • USA001

    RECRUITING

    Columbia, Missouri, 65211, United States

    Contact Email: •••••@•••••

  • USA002

    RECRUITING

    Palo Alto, California, 94304, United States

    Contact Email: •••••@•••••

  • USA004

    RECRUITING

    La Jolla, California, 92037, United States

    Contact Email: •••••@•••••

  • USA006

    RECRUITING

    Philadelphia, Pennsylvania, 19107, United States

    Contact Email: •••••@•••••

  • USA007

    RECRUITING

    Columbus, Ohio, 43221, United States

    Contact Email: •••••@•••••