Drug intelligence / Profile preview

GPX-001

Development stage
Preclinical
Lead developer
Genprex
Modality
Gene Therapies
Administration
Intrapancreatic
01

Overview

GPX-001 is an investigational gene therapy being developed by Genprex for the treatment of Type 1 diabetes. The therapy utilizes an adeno-associated virus (AAV) vector to deliver two key transcription factors, Pancreatic and duodenal homeobox 1 (Pdx1) and MAF BZIP Transcription Factor A (MafA), directly to the pancreas. This approach is designed to facilitate the transdifferentiation or 'reprogramming' of pancreatic alpha cells into functional, insulin-producing beta-like cells. By creating a new source of insulin-producing cells within the patient's own pancreas, GPX-001 aims to restore endogenous insulin production and improve glycemic control, potentially reducing or eliminating the need for exogenous insulin injections. The technology was licensed from the University of Pittsburgh and is currently in preclinical development.

02

Targets

MAFA (V-maf musculoaponeurotic fibrosarcoma oncogene homolog A)PDX1 (Pancreas/duodenum homeobox protein 1)

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