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CPK850

Development stage: Phase 2
Lead developer: Novartis
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Subretinal

Overview

CPK850 is a subretinally delivered gene therapy developed by Novartis for the treatment of retinitis pigmentosa caused by mutations in the retinaldehyde binding protein 1 (RLBP1) gene. The therapy uses an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional copy of the human RLBP1 gene directly to retinal cells. By restoring RLBP1 function, CPK850 aims to improve or stabilize visual function in affected patients. The drug is currently in phase II clinical trials and has not yet received regulatory approval[1][3][5].

Targets

RLBP1 (Retinaldehyde Binding Protein 1)

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