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Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002

Cancer Institute and Hospital, Chinese Academy of Medical Sciences · 2025
ClinicalTrials.gov · Datasets · License: Public Domain · 2025
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This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors. ST002 injection is a gene therapy product designed for NF2. By reinserting the normal XXX gene into genetically deficient tumor cells, the product expresses Merlin. This regulates gene transcription in tumor cells, controls the tumor microenvironment, and inhibits tumor growth and invasion, achieving therapeutic effects.
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