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NOGA-101

Development stage: Preclinical
Lead developer: NOGA Therapeutics
Modality: Gene Silencing → Gene Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies
Administration: Intravenous

Overview

NOGA-101 is a lentiviral vector-based gene therapy developed to treat X-linked agammaglobulinemia (XLA), a rare genetic primary immunodeficiency caused by mutations in the BTK gene. The therapy works by delivering a functional copy of the BTK gene directly into the patient's own stem cells, enabling these re-engineered cells to mature into B-cells capable of producing antibodies. This approach aims to provide a one-time, potentially curative treatment for XLA patients, who otherwise require lifelong antibody replacement and antimicrobial therapies[1][2][3][4].

Targets

BTK (Bruton tyrosine kinase)

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