Drug intelligence / Profile preview

AAV.INS.GLP1

Development stage
Preclinical
Lead developer
Fractyl Health
Modality
Gene Therapies
Administration
Parenteral
01

Overview

AAV.INS.GLP1 is an investigational, pancreas-targeted gene therapy developed by Fractyl Health for the treatment of type 2 diabetes (T2D). It utilizes an adeno-associated virus (AAV) vector to deliver a transgene encoding human glucagon-like peptide-1 (GLP-1) under the regulatory control of an engineered insulin promoter. This approach is designed to achieve durable, beta-cell-specific, and nutrient-responsive GLP-1 expression and secretion directly within the pancreas. By localizing GLP-1 production, the therapy aims to enhance beta-cell secretory function, reduce cellular stress (including oxidative and endoplasmic reticulum stress), and provide long-term glycemic control with a single dose, potentially overcoming the adherence and rebound challenges associated with systemic GLP-1 receptor agonist medications.

02

Targets

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