Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
DNA-based leptin gene therapy
- Development stage
- Preclinical
- Lead developer
- Entos Pharmaceuticals
- Modality
- Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous, Intrathecal
01
Overview
DNA-based leptin gene therapy (DNA-Lep) is a non-viral gene therapy candidate developed by Entos Pharmaceuticals for the treatment of congenital generalized lipodystrophy (CGL), obesity, and other metabolic disorders. The therapy utilizes the proprietary Fusogenix proteolipid vehicle (PLV) platform to deliver a nanoplasmid DNA vector encoding the leptin gene. By facilitating the expression of functional leptin, the therapy aims to restore physiological leptin signaling through the leptin receptor, thereby regulating energy balance, reducing hyperphagia, and improving metabolic parameters such as insulin sensitivity and hepatic lipid accumulation. Preclinical data in rodent models have demonstrated that a single administration (either intravenous or intrathecal) can lead to sustained improvements in body weight and metabolic function, with potential for enhanced efficacy when used in combination with GLP-1 receptor agonists like semaglutide.
- Other names
- PLV-based leptin gene therapy
02
Targets
LEPR (Leptin receptor)
Beyond the preview
Go deeper on DNA-based leptin gene therapy.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect 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 DNA-based leptin gene therapy.