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Emerging Technologies to Rare Disorder Drug Development: Promises and Pitfalls

Feb 25 2014 11:00AM - Feb 25 2014 12:30PM | Online

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This webinar will be broadcast in Eastern Time (ET)

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

Contact Information 

Printable Registration Form

Registration Questions and Technical Support Day of Event
Customer Service Department
Toll Free: 1.888.257.6457
Phone: +1.215.442.6100
Fax: +1.215.442.6199

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

Event Logistics
Colleen Buckley, Event Planner
Fax +1-215-442-6199

Technical Requirements 

Click here for minimum system requirements.

Technical Support Day of Event
Customer Service Department
Phone: +1.888.257.6457 | +1.215.442.6100


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Continuing Education 

The Drug Information Association is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. This program is designated for 1.5 contact hours or .15 continuing education units (CEU’s). 0286-0000-14-039-L04-P; Type of Activity: Knowledge

ACPE Credit Request Update
DIA is required by the Accreditation Council for Pharmacy Education (ACPE) to report pharmacy-requested CEUs through the CPE Monitor system. All ACPE-certified activity credit requests need to be submitted through DIA’s My Transcript within 45-days post activity. Pharmacists will need to provide their National Association of Boards of Pharmacy (NABP) e-Profile ID and date of birth (MMDD) to ensure the data is submitted to the ACPE and NABP properly. If you need to obtain your NABP e-Profile, please visit www.cpemonitor.net.

The Drug Information Association has been accredited as an Authorized Provider by the International Association for Continuing Education and Training (IACET), 1760 Old Meadow Road, Suite 500, McLean, VA 22102; +1.703.506.3275.

As an IACET Authorized Provider, Drug Information Association offers CEUs for its programs that qualify under the ANSI/IACET Standard. Drug Information Association is authorized by IACET to offer .2 CEUs for this program. Participants must attend the entire program in order to be able to receive an IACET statement of credit. No partial credit will be awarded.

This program is part of DIA’s Certificate Program and is awarded the following:
• Clinical Research Certificate Program: 1 Elective Unit
• Clinical Safety and Pharmacovigilance Certificate Program: 1 Elective Unit
• Project Management Certificate Program: 1 Elective Unit
• Regulatory Affairs Certificate Program: 1 Elective Unit

For more information go to www.diahome.org/certificateprograms

Name Credit Type Max Credits CEU
Emerging Technologies to Rare Disorder Drug Develo ACPE 1.50 0.150
Emerging Technologies to Rare Disorder Drug Develo IACET 1.50 0.200

Disclosure Policy:

It is DIA policy that anyone in a position to control the content of a continuing education activity must disclose to the program audience (1) any real or apparent conflict(s) of interest related to the content of their presentation and/or the educational activity, and (2) discussions of unlabeled or unapproved uses of drugs or medical devices. Disclosure statements will be included in the course materials.

Statement of Credit:

If you would like to receive a statement of credit, you must participate in the live webinar, and complete the credit request process through My Transcript. To access My Transcript, please go to www.diahome.org, select “Login to My DIA” and you will be prompted for your user ID and password. Select “My Transcript” (left side bar) and “Credit Request” to process your credit request. Participants will be able to download a statement of credit upon successful submission of the credit request. My Transcript will be available for credit requests on Tuesday, March 11, 2104.

Continuing education credits are available to learners who participate in the live webinar. Continuing education credits are not available for participation in the archived version of the webinar.

To view DIA’s Grievance Policy, please visit the CE page on DIA’s website at www.diahome.org/CE

Registration Fees 

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Member Government Individual
Member Academia Individual
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NonMember Government Individual
NonMember Academia Individual
NonMember Individual
Group Discounts

  • 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.

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