Mayo clinic launches quest for custom drugs for rare genetic diseases

NCT ID NCT05236595

ENROLLING_BY_INVITATION Knowledge-focused Sponsor: Mayo Clinic Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study at Mayo Clinic aims to find people with rare genetic diseases who might benefit from a custom-made drug designed to target their specific genetic change. Researchers will collect samples like blood and skin biopsies to develop and test potential treatments. The goal is to eventually create new therapies for conditions that currently have no good options.

What this could mean

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

Active substance: individualized drug matching per genetic disease
What this could lead to: If successful, this could lead to new personalized treatments for people with rare genetic diseases that currently have no options.
What could go wrong: This is an early-stage research study focused on identifying candidates and developing drugs, not testing them yet. Many potential drugs may not work or be safe.

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.

hereditary disease Undiagnosed Diseases

As listed by the trial registrant

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

RARE GENETIC DISEASE UNDIAGNOSED DISEASES

Contacts and locations

Locations

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States
Mayo Clinic Rochester

Minneota, Minnesota, 55905, United States
Mayo Clinic in Arizona

Scottsdale, Arizona, 85259, United States

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