Gene-Targeted Therapies: Early Diagnosis and Equitable Delivery [Day 2]

Currently, technological developments are offering the hope of new therapies for individuals with rare disease at a pace that has the potential for significantly challenging the existing infrastructure for efficient, effective, and equitable delivery. It is important therefore, to plan and be prepared for these challenges. Changes need to be implemented to improve the process of getting more treatments to more patients, more quickly. Gene-targeted therapies have the ability to provide treatments to multiple disorders rapidly. Gene-targeted therapeutics, such as virus-mediated gene replacement, somatic genome editing and oligonucleotide therapies, directly target the causative molecular defect in genetic diseases. For example, recently discovered gene editing approaches can, in principle, correct approximately 90% of all human disease-causing mutations. As such, these technologies are fundamentally different from most previously developed therapies for individual diseases, in that they are therapeutic platforms that can be developed for individual patients based solely on knowledge of disease-causing mutations, independent of clinical disease. There is also the potential to utilize common vectors and platforms to treat multiple disorders caused by different disease-causing mutations in the same gene or in different genes, affording economies of scale. In contrast to the broad therapeutic potential of gene-targeted therapies across multiple diseases, the US healthcare system,...
Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video