Continuing Education credits are not available for the archived offering. 

This archive will be available for purchase through February 28, 2015.
Archive duration: 1 hour 24 minutes

We are living in one of the most exciting times for biotechnology – as new inventions and paradigms are opening new domains that were not even imagined. With each new technology, there is great promise for rare disease drug development; however, it is important to also examine the possible pitfalls and caveats in leveraging these new technologies. In our webinar, we will discuss four new areas and show specific examples in rare disease drug development that illustrate what factors may result in the success or failure of these endeavors.

Specifically, we will discuss:

1. Genomics has enabled an era of gene-targeted or even mutation targeted therapies. We will discuss the advance in sequencing after the Human Genome Project and how this technology, together with a thoughtful development plan, led to a mutation-specific drug for cystic fibrosis.  
2. Antisense technology has been around for a few decades but only recently has it been successfully translated into a therapy. 
3. Exon skipping uses synthetic antisense nucleotides that causes the pre-messenger RNA to bind in a way to functionally delete an exon from the mRNA. This has been explored for muscular dystrophy and has had its own challenges. 
4. Gene therapy also has been around for a few decades, but some early setbacks has delayed its larger use. Recent development of CRISPR technology may revive this effort and result in new rare disease therapeutics.

We will discuss these four areas for the perspectives of technology development and drug development. We will summarize important lessons learned and general principles as we approach other emerging technologies for rare disorder drug development.