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ST001

Development stage: Preclinical
Lead developer: SciTech Development
Modality: Gene Therapies
Administration: Intravenous

Overview

ST001 is a first-in-class in vivo gene therapy candidate being developed by Santo Therapeutics. It utilizes recombinant lentiviral vector (LV) technology to deliver genetic payloads designed to treat various cancers and rare genetic disorders. The therapy is engineered to reprogram cellular self-protection mechanisms, aiming to provide one-time treatments for lifelong cures. While specific biological targets and indications have not been publicly disclosed, the program is a key component of Santo's pipeline, which focuses on high-unmet-need areas such as brain tumors and thoracic malignancies.

Targets

mTOR (Mammalian target of rapamycin kinase)

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