Drug intelligence / Profile preview

PLX-400

Development stage: Preclinical
Lead developer: Polaryx Therapeutics
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intranasal

Overview

PLX-400 is an early-stage gene therapy developed by Polaryx Therapeutics. It is designed to replace missing lysosomal proteins, aiming to treat CLN2 and CLN3 Batten disease. The company intends to advance this therapy towards an FDA Investigational New Drug (IND) application.

Targets

Neuraminidase

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