Grants
Progetto Loto-Interfering
(Silthera Platform)
The business plan of LOTO Biotech Platform (commercially known as Silthera) focuses on the development of a proprietary technology platform that represents an innovation in drug design. The platform integrates advanced machine learning algorithms to optimize the design of bioactive molecules (prodrugs) based on small interfering RNA (siRNA) technology for the treatment of rare autosomal dominant genetic disorders.
The platform aims to progressively improve the efficacy of these prodrugs while reducing their development and testing costs, ultimately enabling their commercialization to B2B customers, including pharmaceutical companies, Contract Research Organizations (CROs), and biotechnology laboratories.
An additional benefit of the platform lies in the optimization of raw material and energy consumption throughout the drug design and testing process, resulting in lower operating costs and a positive environmental impact.
The business plan has been designed to position LOTO Biotech Platform among the world’s leading developers of siRNA-based therapies targeting a specific subset of rare genetic disorders known as dominant-negative diseases. This innovation is unique in its kind, as it introduces not only a breakthrough in the drug development process but also a revolutionary therapeutic product. Unlike similar prodrugs, the platform’s approach is distinguished by its highly specific mechanism of action: it develops siRNA molecules capable of selectively targeting and eliminating the disease-causing allele while preserving the healthy one, even when the difference between the two consists of a single DNA nucleotide among tens of thousands.
siRNA therapies are designed to silence specific pathogenic messenger RNAs (mRNAs), thereby reducing the expression of harmful proteins and significantly improving the quality of life of patients affected by rare genetic disorders. The platform’s primary focus is on dominant-negative diseases, in which the mutated copy of a gene interferes with the function of the healthy copy, causing disease even when one functional allele is present.
Public Co-Financing
To obtain public funding for the implementation of the project, an application (SSISSI00000018) was submitted under the Smart&Start Italia program, governed by Ministerial Decree of 24 September 2014 (Official Gazette No. 264 of 13 November 2014), as subsequently amended and supplemented.
On 4 December 2025, the request for public co-financing was approved (CUP C16I25002480008 / COR 25414420). The company was awarded a subsidized, interest-free repayable grant with a maximum amount of €1,162,403.13, of which €290,600.78 is non-repayable, corresponding to 80% of the eligible expenses for the implementation of the business plan.
Current Status
The project is progressing in line with expectations and delivering satisfactory results. The activities carried out have enabled the launch and advancement of the LOTO-INTERFERING platform (commercially known as the Silthera Platform), the completion of the initial development phases of Molecule 1, the filing of the related patent protection for the prodrug, the synthesis and experimental validation of the first prodrug candidates, and the initiation of activities related to the non-regulatory preclinical phase and industrial valorization.
The most significant achievement to date has been the demonstration of an integrated and iterative development process: the platform generates candidate molecules, the laboratory tests them, the resulting data are fed back into the model, and the design is subsequently refined. This mechanism directly embodies the innovation supported by the public funding and represents the project’s most important technical advancement compared with the original concept.
The documented progress demonstrates the continuous strengthening of LOTO’s ability to transform the platform into an experimental, patent-protected, and industrially valuable asset.