Official websites use .gov

A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS

A lock ( 🔒 ) or https:// means you've safely connected to the .gov website. Share sensitive information only on official, secure websites.

Need Help

Non-viral gene therapy to treat and prevent atherosclerosis

Award Number: R44HL184837
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/01/2026
PERIOD OF PERFORMANCE END DATE: 02/28/2027

Group Awards By:

View Award Description

Non-viral gene therapy to treat and prevent atherosclerosis - ABSTRACT Adeno-associated virus (AAV) vectors can introduce a non-integrating circular DNA episome capable of persisting in the nucleus of long-lived non-dividing cell types such as muscle, but have a number of drawbacks including high manufacturing cost. Lipid nanoparticles (LNPs) offer numerous advantages over viral vectors, but are unable to efficiently deliver DNA to the nuclei of long-lived non-dividing cells capable of lifelong therapeutic gene expression. After fusing with or disrupting the endosomal membrane, LNPs deliver their contents to the cytoplasm. Whereas mRNA only needs to be delivered to the cytoplasm to be expressed, DNA needs to be delivered to the nucleus. We have discovered a novel strategy to deposit DNA into the nucleus of long-lived non-dividing cell types such as muscle, when that DNA has been delivered to the cytoplasm by LNPs. This approach dramatically enhances the efficiency gene transfer by LNP vectors in muscle. The highest impact application of this technology is a non-viral gene therapy vector based on LNPs for treating and preventing atherosclerotic cardiovascular disease (ASCVD). Under this grant, we will leverage the LNP platform to achieve non-immunogenic gene delivery of a therapeutic protein capable of treating and preventing ASCVD. Under this Fast-Track SBIR grant, we will first demonstrate proof-of-concept in mice in Phase I. Under Phase II, we will optimize non- immunogenic gene transfer via LNPs and LNP formulation, and then demonstrate that our LNP vectors efficiently and non-immunogenically deliver their payload to sustain its stable expression in non-human primates. These studies will facilitate the development of a commercial product capable of treating and preventing ASCVD after a one-time treatment with a non-viral gene therapy vector.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $299,664 )
2026	2026	EMMUNE INC	620 MEMORIAL DR STE W-100	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Cardiovascular Diseases Research	000	1	8/24/2026	NEW	$299,664
														Subtotal = $299,664

Grand Total All Awards = $299,664

Top

All Categories

About

Search

Reports

Data Submission

Award Information

Non-viral gene therapy to treat and prevent atherosclerosis

Award Number: R44HL184837

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/01/2026

PERIOD OF PERFORMANCE END DATE: 02/28/2027

Federal Websites

Department of Health & Human Services

HHS Operating Divisions

HHS Staff Divisions

Download A Document Viewer