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GC304

Development stage: Unknown
Lead developer: GeneCradle
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravenous

Overview

GC304 is a gene therapy drug developed by GeneCradle Inc. It is designed for the treatment of primary hypertriglyceridemia, specifically in patients who have a history of acute pancreatitis. The therapy involves the intravenous delivery of a self-complementary adeno-associated virus serotype 5 (AAV5) vector. This vector carries a codon-optimized lipoprotein lipase (LPL) coding sequence, which is driven by a liver-specific promoter (LP), aiming to restore or enhance LPL activity for improved triglyceride metabolism.

Targets

LPL (Lipoprotein lipase)

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