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nAV-101

Development stage: Preclinical
Lead developer: nVector
Modality: Gene Therapies
Administration: Parenteral

Overview

nAV-101 is an adeno-associated virus (AAV) gene therapy candidate being developed by nVector, a biotechnology company spun out from Iron Horse Diagnostics, for the treatment of Amyotrophic Lateral Sclerosis (ALS). The program utilizes nVector's proprietary AAV platform, which is engineered for the targeted delivery of therapeutic genetic payloads specifically to motor neurons. By focusing on precise delivery to the cells most affected by ALS, nAV-101 aims to address the underlying neurodegenerative pathology of the disease while minimizing off-target effects. The drug is currently in the preclinical stage of development.

Targets

GDNF (Glial cell line-derived neurotrophic factor)GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)

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