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NF1 LNP Gene Therapy
- Development stage
- Preclinical
- Lead developer
- CureAge Therapeutics
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Local
01
Overview
CureAge Therapeutics is developing a lipid nanoparticle (LNP)-based gene therapy for the treatment of neurofibromatosis type 1 (NF1), a genetic disorder characterized by the growth of peripheral nerve tumors, including plexiform neurofibromas. The therapy utilizes LNPs to deliver therapeutic nucleic acids specifically to Schwann cells, which are the primary drivers of tumor formation in NF1. The treatment employs a dual-action mechanism designed to restore functional NF1 gene expression while simultaneously eliminating surrounding tumor cells. Administered via local, minimally invasive delivery to specific anatomical locations, this program aims to address the genetic root of the disease. CureAge Therapeutics was launched in early 2025 as a strategic collaboration between the Children's Tumor Foundation and Deep Science Ventures and the therapy is currently in preclinical development.
- Other names
- LNP-based NF1 gene therapyCureAge NF1 therapy
02
Targets
NF1 (Neurofibromin)
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