News

Santo Therapeutics today announced that its lead program, ST002, has received both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration for the treatment of NF2-related schwannomatosis (NF2-SWN).

The dual FDA designations, granted within a short timeframe, highlight the potential of ST002 to address a significant unmet medical need in a rare and serious genetic disorder and support its continued clinical development.

ST002 is an in vivo gene therapy designed to restore NF2 function through gene delivery. The program is built on Santo’s proprietary 5G-LVV platform, an engineered lentiviral vector system optimized for in vivo gene delivery with enhanced safety features and regulated transgene expression, with the goal of modifying disease progression.

NF2-related schwannomatosis is a genetically driven disease characterized by the development of multiple nervous system tumors, including bilateral vestibular schwannomas, often leading to progressive hearing loss, neurological impairment, and reduced quality of life. Current treatment options remain limited, underscoring a significant unmet medical need.

Orphan Drug Designation provides development incentives including potential market exclusivity, tax credits, and enhanced regulatory support. Rare Pediatric Disease Designation may qualify the sponsor for a Priority Review Voucher (PRV) upon approval, subject to applicable regulatory requirements.

“These dual FDA designations represent an important validation of ST002 and its potential to address a high unmet need in NF2,” said Dr. Luke Zhou, founder of Santo Therapeutics. “We remain focused on advancing ST002 with a strong emphasis on safety, therapeutic efficacy, and long-term clinical value.”

“NF2 is a challenging area with almost no therapeutic options, so advancing gene therapy approaches in this field is both scientifically and clinically meaningful,” said Nick Rodgers, Chairman of the Advisory Board. “ST002 reflects a thoughtful approach to vector design and clinical translation in rare genetic disease.”

About Santo Therapeutics Ltd
Santo Therapeutics is an innovative biotechnology company focused on AI-driven biomanufacturing and the development of next-generation in vivo therapies, including gene therapy and in vivo CAR-T. The company integrates artificial intelligence, virtual cell modeling, viral vector engineering, and scalable manufacturing technologies to enable the development of differentiated therapeutic programs.