Custom gene silencer takes aim at a rare form of ALS

NCT ID NCT07703462

ENROLLING_BY_INVITATION Disease control Sponsor: n-Lorem Foundation Source: ClinicalTrials.gov ↗

First seen Jul 14, 2026 · Last updated Jul 15, 2026 · Updated 1 time

Summary

This study tests a personalized antisense oligonucleotide drug designed for a single person with ALS caused by a specific TARDBP gene mutation. The drug aims to reduce harmful TDP-43 protein and slow disease progression. The participant's clinical function, survival, and biomarkers are monitored over 12 months.

What this could mean

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

Active substance: a personalized antisense oligonucleotide drug called nL-TARDB-002
What this could lead to: If successful, this approach could slow or halt ALS progression in people with this specific genetic cause, offering a targeted treatment option.
What could go wrong: This is an early, single-participant study, so results may not apply to others. The drug may not work or could cause side effects.

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.

amyotrophic lateral sclerosis amyotrophic lateral sclerosis type 10

As listed by the trial registrant

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

AMYOTROPHIC LATERAL SCLEROSIS (ALS)

Contacts and locations

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Locations

Jefferson Health

Philadelphia, Pennsylvania, 19107, United States

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