Drug intelligence / Profile preview

CV-14

Development stage
Preclinical
Lead developer
Cellivery Therapeutics
Modality
Recombinant Proteins and Enzymes
Administration
Injection
01

Overview

CV-14 is an experimental therapeutic candidate developed by Cellivery Therapeutics for the treatment of Friedreich Ataxia (FA), a progressive neurodegenerative disorder. The drug is developed using Cellivery's proprietary Therapeutic Substance Delivery Technology (TSDT) platform, which utilizes Advanced Macromolecule Transduction Domains (aMTD) to facilitate the delivery of large therapeutic proteins across cell membranes and the blood-brain barrier. CV-14 is designed to deliver functional Frataxin (FXN) protein directly into the mitochondria of affected cells. Since Friedreich Ataxia is caused by a genetic deficiency of Frataxin leading to mitochondrial dysfunction and impaired iron-sulfur cluster biogenesis, CV-14 aims to restore these essential cellular processes and mitigate the neurological and cardiac symptoms of the disease.

02

Targets

FXN (Frataxin)

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