Loto Biotech

Loto Biotech is a biotechnology company specializing in the development of RNA-based therapies for rare and genetic diseases, with a specific focus on dominant negative autosomal disorders. The company was founded by Mattia Capulli, Isabella Baldini, and Riccardo Paone, who collectively possess extensive experience in siRNA technology and the regulatory approval processes for orphan drugs.

The core of the company's operations is its proprietary platform that designs and develops small interfering RNA (siRNA) molecules. This technology is engineered to selectively "silence" or switch off the mutated genes responsible for specific rare genetic diseases while leaving the healthy corresponding gene unaffected. This approach has been applied to conditions such as autosomal dominant osteopetrosis type 2 and megacystis-microcolon-intestinal hypoperistalsis syndrome (MMIHS). The platform's methodology aims to reduce drug development times by 50% and costs by 25% compared to traditional methods. Its modular nature also allows for scalability across hundreds of different diseases, with potential applications in oncology, cardiovascular, and metabolic disorders.

Headquartered in L'Aquila, Italy, within the Tecnopolo d'Abruzzo, Loto Biotech operates in the biotech and HealthTech sectors. In June 2025, the company secured €600,000 in funding from Scientifica Venture Capital. This investment provides capital and access to Scientifica Lab, a 4,000 m² facility designed to accelerate the transition from research concepts to prototypes and support the preclinical discovery process.

Keywords: siRNA therapies, rare genetic diseases, gene silencing, orphan drugs, autosomal dominant disorders, drug development platform, biotechnology, healthtech, precision medicine, RNA-based therapeutics, preclinical discovery, genetic mutations, molecular biology, personalized medicine, venture capital, Scientifica Venture Capital, Mattia Capulli, Isabella Baldini, Riccardo Paone, Tecnopolo d'Abruzzo