Therapy to clear harmful protein clumps in Alzheimer's, FTD, and ALS

Developing an Anti-proteotoxicity Therapy for Neurodegenerative Diseases

['FUNDING_OTHER'] · REZIGENE, LLC · NIH-11194249

Developing small-molecule medicines that help brain and nerve cells remove harmful misfolded proteins in people with Alzheimer's disease, frontotemporal dementia, or ALS.

Quick facts

Phase['FUNDING_OTHER']
Study typeNih_funding
SexAll
SponsorREZIGENE, LLC (nih funded)
Locations1 site (BALTIMORE, UNITED STATES)
Trial IDNIH-11194249 on ClinicalTrials.gov

What this research studies

This project looks for new molecular targets and small medicines that boost cells' natural systems for handling misfolded proteins. Scientists use large, unbiased lab screens along with genetic, biochemical, and cell-based tests to find and refine compounds that reprogram protein quality control. The work explores pathways beyond the classic heat shock or unfolded protein responses to better prevent toxic protein clumping. Promising compounds would be advanced toward further preclinical testing and eventual clinical studies.

Who could benefit from this research

Good fit: People diagnosed with Alzheimer's disease, frontotemporal dementia, or amyotrophic lateral sclerosis—especially those in earlier stages and willing to join future clinical trials—would be the likely candidates.

Not a fit: People without these neurodegenerative diseases, or those in very advanced stages with widespread damage, may not receive benefit from this approach.

Why it matters

Potential benefit: If successful, the therapy could reduce toxic protein aggregates and slow nerve-cell damage, potentially slowing symptom progression in Alzheimer's, FTD, or ALS.

How similar studies have performed: Earlier lab and animal studies targeting proteostasis have reduced protein aggregates, but translating those findings into proven human therapies for common neurodegenerative diseases remains largely unproven.

Where this research is happening

BALTIMORE, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Conditions: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease, Amyotrophic Lateral Sclerosis Motor Neuron Disease

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.