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NOGA-201

Development stage: Preclinical
Lead developer: NOGA Therapeutics
Modality: Gene Therapies
Administration: Parenteral

Overview

NOGA-201 is an *in vivo* hematopoietic stem cell (HSC) lentiviral gene therapy being developed by NOGA Therapeutics for the treatment of X-linked agammaglobulinemia (XLA). It utilizes the company's proprietary Lent-Me-In platform to deliver a therapeutic Bruton's tyrosine kinase (BTK) expression cassette directly to HSCs within the patient's body. This "off-the-shelf" approach aims to restore BTK function and B-cell development without the need for the complex *ex vivo* cell processing and conditioning regimens typically associated with traditional HSC gene therapies. NOGA-201 shares the same therapeutic cassette as NOGA-102, which is an *ex vivo* autologous HSC gene therapy candidate.

Targets

BTK (Bruton tyrosine kinase)

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