Drug intelligence / Profile preview

KNA-165

Development stage: Preclinical
Lead developer: Kinea Bio
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravenous

Overview

KNA-165 is an investigational gene therapy being developed by Kinea Bio for the treatment of Congenital Muscular Dystrophy Type 1C (MDC1C). MDC1C is a rare, severe neuromuscular disorder caused by mutations in the fukutin-related protein (FKRP) gene, which leads to the hypoglycosylation of alpha-dystroglycan and subsequent muscle membrane instability. KNA-165 is designed to deliver a functional copy of the FKRP gene to muscle cells, potentially restoring the glycosylation of alpha-dystroglycan and improving muscle function and stability. The program is currently in the early stages of development.

Targets

FKRP (Fukutin-related protein)

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