On March 20, 2025 Santo Therapeutics, a biotechnology company focused on advancing gene therapies, reported the initiation of its first Investigator-Initiated Clinical Trial (IIT) in China for ST002. Santo’s ST002 is an innovative in vivo lentiviral gene therapy designed to treat tumours with NF2 gene mutations. This marks a major milestone as ST002 is the world’s first gene therapy targeting NF2 mutation-driven tumours to enter clinical trials.
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"We are excited to start this groundbreaking clinical trial," said Dr. Luke Zhou, CEO of Santo Therapeutics. "ST002 is the first gene therapy specifically targeting NF2-related schwannomatosis (NF2-SWN), and with our proprietary platform, we are introducing a mechanism that precisely targets both the tumour and its environment, providing a more effective solution for NF2-related tumours."
Nick Rodgers, Chairman of the Advisory Board of Santo, commented, "Currently, there is no cure for NF2 disease. Gene therapy is the only promising solution to offer a cure, and we are eager to see the potential of ST002. Having spent months in pre-clinical testing we are pleased to be working with The Cancer Institute and Hospital, Chinese Academy of Medical Sciences to validate our therapy in humans."
The first-in-human IIT for ST002 is now underway, with initial clinical data expected to be available in the second half of 2025. In preclinical studies, ST002 demonstrated notable anti-tumour effects in NF2 patient-derived-xenograft (PDX) models, significantly inhibiting tumour growth and showing high specificity and durability.
"We are excited to advance ST002 to the clinical stage and bring hope to patients suffering from tumours with NF2 mutations," Dr. Zhou added. "This therapy holds the potential to not only treat NF2-related tumours but also open new avenues for treating other rare genetic conditions. We look forward to sharing our progress and early results in the coming months."
About ST002
ST002 is an innovative lentiviral gene therapy that delivers precise genetic material directly to the tumour site. By targeting the tumour micro-environment and restoring NF2 gene function, ST002 inhibits tumour growth by blocking abnormal Schwann cell. As an off-the-shelf product, ST002 offers a cost-effective, scalable treatment option for patients with schwannoma and other NF2 related tumours.
(Press release, Santo Therapeutics, MAR 20, 2025, View Source [SID1234663856])