Nucleic Acid Therapeutics: Rationale, Approaches, Progress and Challenges

Industry Short Course
May 2-4, 2011
Pyle Center, University of Wisconsin
702 Langdon Street
Madison, WI


On Campus Short Courses for the Pharmaceutical Industry



Goals and Objectives

The purpose of this short course is to provide an introduction to various classes of nucleic acid therapeutics, including gene therapy, antisense oligonucleotides, microRNA, and siRNA. The course will cover the scientific, development, manufacturing, regulatory, and intellectual property aspects of commercializing these novel therapeutics. Further, the topic of nucleic acid therapeutic delivery, including assessment of various approaches taken as well as their successes and remaining challenges will be discussed. Learners will be given an opportunity to work through case studies and apply the foundations for nucleic acid therapeutics to the challenges of bringing these novel entities to first in man clinical studies.

Upon completion of this course, the learner should be able to:

  1. Short courseDescribe various classes of nucleic acid therapeutics and their relative attributes for disease management or treatment.
  2. Describe the mechanism of action for the various classes of nucleic acid therapeutics.
  3. Recognize the challenges of delivering these therapeutics to their intended sites of action and describe the approaches being used and developed.
  4. Discuss the various regulatory issues involved in nucleic acid drug development.
  5. Identify key aspects of a potential therapeutic that will determine its feasibility in manufacturing, delivery and efficacy.
  6. Describe the legal and intellectual property issues surrounding the science, production, and delivery of nucleic acid therapeutics.
  7. Apply principles learned to case simulations.

^ Back to menu

General Information


The primary objective in all short courses is to upgrade the knowledge of pharmaceutical personnel, who can then be of greater value to their respective firms. Students will work closely with one or more faculty members to allow for optimum dialogue and discussion. We also have modified some of the courses based on evaluations and suggestions from previous participants. As with all our programs, these are designed to be practical and applicable to participants' work environments. To allow for maximum faculty-student interaction, the enrollment will be limited and available to learners on a first-come basis.

Last year all attendees strongly agreed that the program was a valuable learning experience and all indicated that they would recommend the course to a colleague.


Courses are presented on the University of Wisconsin-Madison Campus. Springtime in Madison and the beauty of the on-campus location provide a relaxing atmosphere for learning. Evenings can be used to explore the University and the city named by Money magazine as the "best place to live in America" in 1996. For more information about Madison, WI visit

Madison, WI



Housing information will be sent to individuals when they receive their registration confirmation. Several hotels are within a short walking distance from the conference facility. The approximate cost per night is $130.

Ending / Starting Times

The course begins at 8:30 AM on Monday and ends at approximately 11:00 to 11:30 AM on the last day of the class. This allows participants to make reservations for return flights the same day. This allows participants to make reservations for return flights the same day.

Enrollment & Fees

Unless otherwise indicated, enrollment will be limited to 30 individuals per short course. The registration fee of $1095.00 includes instructional costs, course materials, and coffee breaks, and lunches. Cancellations after April 15 are non-refundable. Cancellations prior to April 15 are subject to a $75 administration fee. In the event that an accepted applicant cannot attend, a substitute may be sent by the same company.

Early bird rate ($845) is offered to individuals registering prior to April 15, 2011. You are encouraged to register early to avoid not being able to be included in the program.

Special Offer for Academic Institutions
Extension Services in Pharmacy at the University of Wisconsin School of Pharmacy has a policy for industrial professional development programs to encourage greater participation by members of academic institutions. Programs will be offered at a significantly reduced price, based on space availability. For information about any specific course please contact

^ Back to menu

Course Contents

  • Overview and history of nucleic acid therapeutics
  • Gene therapy
    • Vectors for transgene expression, advantages and disadvantages
      • Plasmid DNA
      • Viral vectors: AAV, Lentivirus, Adenovirus, others
    • Delivery methods for pDNA and target tissues
      • Naked pDNA
        • Electroporation
        • Intramuscular injection
        • Hydrodynamic injection
      • Nanoparticles, polymers, lipids
    • Translation of therapy from animal models to the clinic
      • Efficacy studies
      • Limitations of the animal model
        • Scaling-up
        • Biological differences
      • Safety studies
        • Biology: antibody and innate immune responses
        • Toxicity of vector, delivery method, and reagents in non-human primates
    • Challenges of administrating gene therapy to humans
      • Manufacturing and quality control
      • Regulations
  • RNA interference, antisense, aptamers and immunostimulatory oligonucleotides
    • Overview and history
    • Mechanisms of action
    • Delivery approaches
      • Polymers, lipids, molecular conjugates, chemical modifications
    • Challenges to reach the clinic: key issues in development
      • Proof-of-concept/Pharmacology studies
      • Preclinical safety and efficacy models
      • Regulatory expectations: Preclinical studies
      • Regulatory expectations: Chemistry, Manufacturing and Controls
  • · Intellectual property issues

^ Back to menu


Jeremy Heidel, Ph.D.
Independent Consultant, Madison, WI

Rebecca B. Scarr, J.D., Ph.D.
Attorney at Law, Andrus, Sceales, Starke & Sawall, LLP, Milwaukee, WI

G. Susan Srivatsa, Ph.D.
President, ElixinPharma, Encinitas, CA

Raghu Vemuganti, PhD
Associate Professor, Department of Neurological Surgery
University of Wisconsin, Madison WI

Christine Wooddell, Ph.D.
Senior Scientist, Roche Madison Inc., Madison, WI

Michael V. Templin, Ph.D., DABT
VP, Research and Development, MDRNA Inc. Bothell, WA

^ Back to menu

Contact Us

If you should have questions or require more information, please contact:

Eric Buxton, Ph.D.
Course Coordinator
Extension Services in Pharmacy
777 Highland Avenue
Madison, WI 53705
(608) 262-2431 FAX
(608) 262-3130

^ Back to menu