Creating new lipid nanoparticles to deliver genetic material to the brain

Development of novel lipid nanoparticles for nucleic acids delivery in the brain

['FUNDING_U01'] · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · NIH-11095264

This study is exploring a new way to help deliver important treatments directly to brain cells using tiny particles that can safely cross into the brain, which could be especially helpful for people with Alzheimer's disease.

Quick facts

Phase['FUNDING_U01']
Study typeNih_funding
SexAll
SponsorICAHN SCHOOL OF MEDICINE AT MOUNT SINAI (nih funded)
Locations1 site (NEW YORK, UNITED STATES)
Trial IDNIH-11095264 on ClinicalTrials.gov

What this research studies

This research focuses on developing lipid nanoparticles (LNPs) that can effectively deliver nucleic acids, such as RNA, directly to brain cells. These nanoparticles are designed to cross the blood-brain barrier, which is a significant challenge in treating neurological disorders. By engineering these LNPs to be biocompatible and biodegradable, the research aims to enhance the stability of the nucleic acids and ensure their safe delivery to targeted brain regions. The study will utilize various animal models to test the effectiveness of these nanoparticles in delivering therapeutic agents to neurons affected by conditions like Alzheimer's disease.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals diagnosed with Alzheimer's disease or related neurodegenerative conditions.

Not a fit: Patients with non-neurological conditions or those who do not have a diagnosis related to Alzheimer's disease may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective treatments for Alzheimer's disease and other neurological disorders by enabling targeted delivery of genetic therapies to the brain.

How similar studies have performed: Previous research has shown promise in using lipid nanoparticles for drug delivery, indicating a potential for success in this novel application for brain therapies.

Where this research is happening

NEW YORK, 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

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.