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Emerging Technologies to Rare Disorder Drug Development: Promises and Pitfalls


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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.

Featured topics

  • Orphan diseases
  • Rare disorders
  • Early clinical development
  • Pharmacokinetic/ pharmacodynamics modeling
  • Clinical trials
  • Genomics
  • Targeted therapeutics
  • Antisense therapeutic
  • Gene therapy
  • Exon skipping
  • Cystic fibrosis
  • Muscular dystrophy

Who should attend?

  • Academic researchers
  • Pharmaceutical/Biotechnology clinical development groups (Therapy Area Heads, Operations and Project Management, Regulatory, Clinical Safety and Pharmacovigilance)
  • Patient organizations
  • Investors focused on the future of orphan product development
  • Policy experts concerned about federal or state policies that affect patients with rare diseases
  • Providers of services to the rare disease community, including insurance providers and health care professionals
  • Government officials responsible for rare disease research and orphan product oversight
  • Reimbursement specialists
  • Statisticians
  • Pharmacologists
  • Biologists
  • Clinical scientists
  • Human geneticists

Learning objectives

At the conclusion of this webinar, participants should be able to:

  • Discuss new genomics technologies and the development of mutation-targeted therapies
  • Describe antisense technologies and its use as a therapeutic
  • Explain exon skipping and how the translation of this technology is being explored for use in muscular dystrophy
  • Discuss gene therapy, its history context, and new promise with CRISPR technology

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Agenda Details
Carrie Dunn, Content Lead
Phone +1.215.442.6181
Fax +1.215.442.6199

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Colleen Buckley, Event Planner
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Ways to register

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

  • Individual registration is a license for ONE internet login allowing one viewer.
  • Group site registration is a license for ONE internet login allowing multiple viewers from one location.
  • Group Plus is a license for up to FIVE internet logins allowing one or more viewers at each login location.

Cancellations:  No refunds will be provided in the event of a participant’s cancellation since all costs for this webinar have been prepaid by DIA. DIA reserves the right to modify or cancel programs and/or substitute presenters or panelists. DIA is not responsible for failure to deliver programs due to circumstances beyond its control.

Participants with Disabilities: Reasonable accommodations will be made available to persons with disabilities who attend an educational activity. Contact the DIA office in writing at least 15 days prior to event to indicate your needs.