The Platform
The LOTO siRNA interacts with mutated genes to restore normal functionality or improve the disease synthoms. The mechanism involves the targeted silencing of the mutated allele by the administered siRNA treatment, thereby removing its deleterious effects. In this state, only the healthy allele serves as a template for protein synthesis, ensuring that the produced proteins are normal and not mutated.
The LOTO Biotech Platform
At our company, we leverage proprietary algorithms to identify the optimal targets for our drugs. Our development plan is focused on three specific diseases, chosen based on several critical criteria: a high unmet need, a relatively low threshold for clinical success, objectivity of clinical results, risk reduction in our portfolio through transferability to other indications, and relatively low CAPEX requirements. By prioritizing these factors, we aim to maximize the impact and efficiency of our therapeutic developments.
Our Drug Candidates
Osteogenesis Imperfecta (OI)
Osteogenesis Imperfecta (OI), also known as brittle bone disease, is a rare genetic bone disorder impacting skeletal health, breathing, and swallowing. The condition primarily results from dominant mutations in the COL1A1 and COL1A2 genes, accounting for 90% of cases. Children with the severe Type III form of OI have a life expectancy of around 10 years. This debilitating disorder affects approximately 49,000 individuals in the US and EU5, highlighting the urgent need for effective treatments and support for those living with OI.
Megacystis Microcolon Hypoperistalsis Syndrome
Our in vitro treatment for Megacystis Microcolon Hypoperistalsis Syndrome (MMIHS) utilizes our advanced platform to identify the best candidates for addressing the phenotype associated with the ACTG2 gene mutation c.532C>T (protein mutation p.Arg178Cys or p.R178C). Using HEK293 cell models carrying the mutation, we rapidly screened all candidates and identified the top molecule (Molecule C), which shows maximum interaction with the mutated gene and minimal interaction with the wild-type allele. While we are developing a murine model for pre-clinical trials, we have also prioritized engaging with the EMA. Through several preliminary meetings, we aim to expedite the exploration of new experimental avenues, especially where time is limited and animal models and patient cohorts for testing are scarce or non-existent.
Marfan Syndrome
Marfan syndrome is a genetic disorder affecting the body's connective tissue, leading to severe complications in the musculoskeletal and cardiovascular systems, as well as the eyes. With proper medical and surgical intervention, diagnosed patients have an average life expectancy of 50 years. However, severe cases have a significantly reduced life expectancy of only 5 years. Notably, 85% of Marfan syndrome cases are caused by dominant mutations in the KRT5 gene. This condition also manifests as "butterfly skin," characterized by significant blistering, scarring, and infections in open sores, affecting approximately 17,000 individuals in the US and EU5.