The LOTO Biotech Platform

LOTO Biotech Platform is an innovative biotech startup specializing in the development of RNA-based therapies for the treatment of rare and genetic diseases, with a particular focus on dominant negative autosomal disorders. LOTO positions itself as a reference point in the sector thanks to its cutting-edge technology and the expertise of its team.

There are approximately 7,000 confirmed rare genetic diseases, and numerous genetic variants specific to each pathology add to this number, affecting each patient uniquely. Our proprietary platform can predict siRNA sequences capable of specifically silencing messenger RNAs that carry pathological mutations, thereby reducing the expression of harmful proteins and minimizing side effects. This technology has demonstrated its effectiveness in conditions such as autosomal dominant osteopetrosis type 2 and megacystis-microcolon-intestinal hypoperistalsis syndrome (MMIHS).

The three founders of LOTO Biotech boast many years of experience both in the application of siRNA technology and in navigating the regulatory approval processes for orphan drugs with regulatory bodies such as the EMA. This combination of technical and regulatory expertise allows us to successfully tackle market challenges.

With an innovative approach and a clear vision, LOTO Biotech Platform is committed to transforming the treatment of rare genetic diseases, offering new hope to patients worldwide.