Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
AAV-NFE2L1
- Development stage
- Preclinical
- Lead developer
- Harvard T.H. Chan School of Public Health
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV-NFE2L1 is a preclinical, research-stage gene therapy designed to deliver the wild-type NFE2L1 (nuclear factor erythroid 2-related factor 1) gene using an adeno-associated viral (AAV) vector. Developed at the Harvard T.H. Chan School of Public Health, the therapy targets the hepatic NFE2L1/INSIG1 pathway, which acts as a control switch for cholesterol and lipid homeostasis in liver cells. In preclinical models of metabolic dysfunction-associated steatohepatitis (MASH), NFE2L1 overexpression has been shown to activate SREBP1/2, lower hepatic cholesterol, and attenuate liver injury, inflammation, and fibrosis without elevating atherogenic lipoproteins. The therapy is being investigated for the treatment of MASH-associated atherogenic dyslipidemia.
- Other names
- AAV-NFE2L1-WTAAV-NFE-2L1-WTAAV-NFE 2L1-WT
02
Targets
INSIG1 (Insulin-induced gene 1 protein)SREBP-1 / SREBF1 (SREBP-1)SREBP-2 / SREBF2 (SREBP-2)
Beyond the preview
Go deeper on AAV-NFE2L1.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on AAV-NFE2L1.