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LX2001

Development stage: Unknown
Lead developer: Lexeo Therapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration: Subretinal

Overview

LX2001 is an investigational gene therapy developed by Lexeo Therapeutics for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. It is designed as an adeno-associated virus (AAV)-based gene therapy intended to deliver a functional copy of the RPGR gene to retinal cells, aiming to restore or preserve vision in affected individuals. The therapy is administered via subretinal injection and represents a targeted approach for this inherited retinal degenerative disease. As of 2025, LX2001 remains in clinical development.

Targets

RPGR (Retinitis pigmentosa GTPase regulator)

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