Workshop: In Silico Drug Design and Repurposing for Rare Diseases

Among the 7,000 identified rare diseases, only 5% of these conditions have an approved drug. The challenges in developing treatments for rare diseases stem from limited market size and high development costs, deterring pharmaceutical companies from investing in research and development. Consequently, patients with rare diseases often face delays or lack of access to life-saving treatments, highlighting the need for collaborative efforts to incentivize and accelerate innovative therapy development. Next-generation sequencing has revolutionized genetic testing, enabling the identification of disease-causing gene mutations essential for precision medicine implementation. Understanding the genetic and molecular mechanisms of rare diseases is essential for identifying therapeutic targets, a task facilitated by computational techniques. These methods leverage advanced algorithms and bioinformatics tools to analyse genomic and chemical data, accelerating drug discovery and repurposing efforts. Ultimately, these approaches hold promise for advancing precision medicine and improving outcomes for individuals affected by rare genetic diseases. This AGYA Workshop offers an Arab-German platform for exchange on the latest research results.