LOTO Biotech Platform srl (trademark Silthera)

Silthera is a biotechnology company developing precision RNA therapeutics for rare dominant-negative genetic diseases. Its proprietary TRL 7 platform combines artificial intelligence, machine learning, thermodynamic modelling and wet-lab validation to identify and prioritise allele-selective siRNA candidates before synthesis. By integrating computational prediction with experimental feedback, Silthera aims to reduce development iterations, shorten timelines and lower early R&D costs.

The company focuses on diseases where selective silencing of the mutated allele, while preserving the healthy allele, is essential to achieve a clinically meaningful effect. Silthera currently has three patent-pending therapeutic programmes in visceral myopathy and osteogenesis imperfecta and operates its own R&D laboratory in Italy.

Silthera’s model sits between academic research and pharmaceutical development: it translates scientific discoveries into de-risked, licensable therapeutic programmes through an integrated approach combining biology, AI-driven design, experimental validation, regulatory strategy and industrial execution. The company works with specialised scientific and development partners while retaining ownership of its core platform and therapeutic assets.

Silthera’s long-term ambition is to make precision RNA therapeutics scalable and economically viable for rare genetic diseases that are often underserved by traditional drug-development models, ultimately helping promising science progress closer to the patients who need it most.