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DIA/FDA Oligonucleotide-based Therapeutics Conference

Sep 25 2013 8:00AM - Sep 27 2013 5:00PM | Washington Court Hotel 525 New Jersey Avenue NW Washington, DC 20001

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Overview 

CALL FOR POSTER ABSTRACTS

DIA/FDA Oligonucleotide-based Therapeutics Conference
Submit a Poster Abstract

SUBMISSION DEADLINE: Monday, July 1, 2013
Read Poster Submission Details and Guidelines 



For the fifth time in seven years, DIA and FDA have convened industry and health authorities to inform, educate, and share advancements in oligonucleotide-based therapeutic product development. The conference will incorporate dialogue between regulators and industry from CMC, Nonclinical, Clinical Pharmacology, and Clinical disciplines to address the developmental advances, safety, and challenges in the field of oligonucleotide-based therapeutics. The 2013 event will address quality risk management, manufacturing advances, specifications, formulation issues, CMC strategies, oligonucleotide pharmacokinetics, nonclinical assessments in support of drug development and clinical advances in therapeutic targets, trial design and safety for antisense, siRNA and microRNA therapies. In addition, the sessions will cover a wide range of current topics in oligonucleotide science and feature expert speakers from industry and regulatory agencies.  Each session will consist of presentations and panel discussions in an interactive format designed to promote discussion between industry and the regulators ending with a session on emerging hot topics in oligonucleotide-based therapeutics.

 

Featured Topics 

Nonclinical Track
The nonclinical sessions are designed to provide updates and discussion on recent advancements in nonclinical development of oligonucleotide therapeutics. The track will address emerging approaches for development of oligonucleotide therapeutics with focus on the predictivity of animal models and the applicability of ICH guidelines to specific concerns in development. Regulatory challenges in clinical pharmacology, clinical pharmacology assessment, and approaches to animal-to-human dose scaling will be presented. The specific area of oligonucleotide immunogenicity assessment will be examined. The nonclinical track will also join with the clinical track in a combined session discussing the relevance of nonclinical toxicities in kidney to findings in clinical trials. This session is intended to address the safety assessment practices, diverse developmental challenges, and emerging potential of oligonucleotide therapeutics.

  • Updates and Future Directions for the Oligonucleotide Safety Working Group (OSWG)
  • Clinical Pharmacology Assessment and its Regulation
  • Approaches to Immunogenicity Assessment for Oligonucleotide Therapeutics
  • Regulatory Experience Including Discussion of Dose Scaling, and the Influence of Structure and Sequence on Oligonucleotide Toxicity
  • Kidney Toxicities: Translational Relationship of Nonclinical Data to Clinical Findings
  • Hot Topics Including Novel miRNA Contributions to Disease and Therapy

Clinical Development Track
These sessions will provide updates on the recent progress made with oligonucleotides in the clinic.  Programs in various stages of development will highlight the challenges faced, lessons learned, and offer potential solutions and innovative ideas for clinical development of oligonucleotide therapeutics. Local and parenteral routes of administration of simple and complex formulations will be featured, with corresponding focus on the safety and tolerability of this class of compounds in humans. Talks will span programs ranging from early Phase I to late-stage Phase III. 
Therapeutic areas to be covered will include:

  • Metabolic Disease
  • Oncology
  • Liver Targets
  • Infectious Diseases
  • Kidney Disease
  • Neuromuscular Conditions

Chemistry, Manufacturing, and Controls (CMC) Track
The CMC track will cover a wide range of current oligonucleotide science and feature expert speakers from industry and regulatory agencies. Each session will comprise presentations and a panel discussion in an interactive format designed to promote dialogue between industry and the regulators.
The following topics will be discussed:

  • The Role of Quality Risk Management in Oligonucleotide Drug Development
  • Advances in Oligonucleotide Manufacturing and Control Strategies
  • Analytical Advancements and Oligonucleotide Specifications
  • Novel Excipients and Other Recent Advances in Formulation Development
  • CMC Strategy and Summary of Regulatory Interactions for Mipomersen

Who Should Attend 

Chief Scientific Officers, Vice Presidents, Directors, Senior Management, Group/Team/Project Leaders, Scientists, Investigators and Researchers working in the following areas of oligonucleotide science:

  • Biotechnology
  • Clinical Pharmacology
  • Clinical Research
  • Chemistry, Manufacturing and Control
  • Clinical, Regulatory, and Business Development
  • Delivery Technologies
  • Drug Discovery
  • Preclinical
  • Quality Assurance
  • RNAi
  • Vaccines

Learning Objectives 

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

  • Identify accomplishments and challenges in the clinical development of oligonucleotide-based therapeutic drugs
  • Describe the critical issues in the nonclinical development of oligonucleotides
  • Differentiate the chemistry, manufacturing and controls challenges associated with the development of synthetic oligonucleotides, including formulation and specification issues
  • Explain unique aspects and various scientific approaches used during the development of oligonucleotide-based therapeutics
  • Recognize the achievements made in the field to date and be able to share the vision with patients about the therapeutic potential that oligonucleotides possess across a wide range of indications
  • Discuss industry and regulatory agency efforts to partner and address the unmet medical needs of patients

 

Hotel & Travel 

The conference will be held at the Washington Court Hotel.  A limited number of rooms are available at the reduced rate shown below (DIA rate is guaranteed until September 4, 2013, or until room block is filled). Attendees can follow this link to make their hotel reservations, or by calling +1.212.532.1660 or in the USA at 1.800.221.3531. When making your hotel reservation by phone, please select option 1 for “Hotel Reservations,” inform the phone agent that it is a DIA event, and provide them with the date and title of the meeting.

Please Note: In order to receive the reduced room rate, hotel reservations must be made as noted above and not directly with the hotel.

Standard Room Rate $259

Hotel Address:  525 New Jersey Avenue NW, Washington, DC 20001

The most convenient airport is Ronald Reagan Washington National Airport (DCA) and attendees should make both airline and hotel reservations as early as possible.

Contact Information 

Registration Questions
Elizabeth Espich, Customer Service Associate
Phone +1.215.293.5802
Fax +1.215.442.6199
Elizabeth.Espich@diahome.org

Agenda Details
Colleen Braun, Content Lead
Phone +1.215.442.6160
Fax +1.215.442.6199
Colleen.Braun@diahome.org

Event Logistics
JoAnn Boileau, Event Planner
Phone +1.215.442.6175
Fax +1.215.442.6199
JoAnn.Boileau@diahome.org

Continuing Education 

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.

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 1.5 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.

CERTIFICATE PROGRAM

This program is part of DIA’s Certificate Program and is awarded the following:
• Clinical Research Certificate Program: 10 Elective Units

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

Name Credit Type Max Credits CEU
DIA/FDA Oligonucleotide-based Therapeutics Confere IACET 15.25 1.500

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 attend the program and tutorial(s), if applicable, sign-in at the DIA registration desk each day of the program, and complete the on-line credit request process through DIA’s My Transcript. No partial credit will be awarded. 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 Friday, October 11, 2013.

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

Program Committee 

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Agenda  

Day 1 Wednesday, September 25, 2013

  • 8:30AM - 8:45AM

    Welcome and Opening Remarks

    Speaker(s):

    • Robert Dorsam, PhD
      Pharmacologist, CDER
      FDA, United States
    • Jim Zisek
      Director, Global CMC Regulatory Affairs
      GlaxoSmithKline, United States
  • 8:45AM - 10:00AM

    Keynote Address

    Speaker(s):

    • Keynote Address
      Arthur A. Levin, PhD
      Executive Vice President R & D
      miRagen Therapeutics, United States
  • 10:00AM - 10:30AM

    Refreshment Break
  • 10:30AM - 12:00PM

    Session 1A (Nonclinical): OSWG Summaries and the Path Forward


    Session Chair(s):

    • Doug Kornbrust, PhD
      consultant
      Preclinsight, United States
    • Session Chair Invited
      FDA, United States

    The Oligonucleotide Safety Working Group (OSWG) is an organization of industry and health authority professionals dedicated to advance the development of oligonucleotides. Included in this session will be OSWG summaries of recent activities and discussion from the OSWG Reproductive and Carcinogenic Risk Subcommittee and the Genetic Toxicology Subcommittee. The session will end with a panel discussion about future organizational and investigative directions for OSWG.

  • 10:30AM - 12:00PM

    Session 1B (CMC): Role of Quality Risk Management in Oligonucleotide Drug Development


    Session Chair(s):

    • James V. McArdle, PhD
      President
      McArdle & Associates LLC, United States

    Risk Management is the identification, assessment, prioritization, mitigation and communication of risk, which is defined as the probability of occurrence and severity of harm. Risk Management has recently become more widely adopted in the pharmaceutical industry, where it is now recognized as an integral component of an effective Quality System. Scientific advice regarding the application of Risk Management to the manufacture and use of pharmaceuticals is provided in ICH guideline: Quality Risk Management, Q9. This session will feature two presentations. The first presenter will summarize the general principles of Quality Risk Management delineated in Q9; the second presenter will discuss the specific application of some of the principles to oligonucleotide drug development. The presentations will be followed by a 30 minute panel discussion.

  • 10:30AM - 12:00PM

    Session 1C (Clinical Development): Neuromuscular


    Session Chair(s):

    • Representative Invited
      FDA, United States
    • Stephen B. Shrewsbury, MD,FFPM
      Senior Vice President, Clinical Development and Chief Medical Officer
      Aquinox Pharmaceuticals Inc., Canada

    This session will review the encouraging emerging clinical data generated over the last 18 months with therapeutic oligonucleotides in neuromuscular disease, where splice switching using the alternative splicing pathway is emerging as a natural process to be harnassed by modern medicines.

  • 12:00PM - 1:30PM

    Lunch
  • 1:30PM - 3:00PM

    Session 2A (Nonclinical): Oligonucleotide Clinical Pharmacology


    Session Chair(s):

    • Richard Stephen Geary, PhD
      Senior Vice President, Development
      Isis Pharmaceuticals, Inc., United States
    • Representative Invited
      FDA, United States

    The development of oligonucleotide therapeutics requires pharmacokinetic and clinical pharmacology assessments that provide a framework for regulatory decisions. This session will focus on the clinical pharmacology of oligonucleotide products, recommended assessments, and current regulation. Presentations include one from the FDA addressing current regulatory practices governing the clinical pharmacology of oligonucleotides and a presentation reviewing clinical pharmacology decisions in the early development of Mipomersen. The session will continue with two complimentary presentations, one from Pharma and one from the FDA discussing real-world measurements and pertinent factors to consider in the important calculation of animal-to-human dose scaling.

  • 1:30PM - 3:00PM

    Session 2B (CMC): Advances in Oligonucleotide Manufacturing and Control Strategies


    Session Chair(s):

    • Steven Broadbent
      Vice President of Quality
      NITTO DENKO Avecia Inc., United States

    It is widely recognized that process and product knowledge comprise important components of any strategy designed to assure the quality of pharmaceutical products. The first presenter will discuss recent advances in understanding the chemistry of solid phase oligonucleotide synthesis through the application of process analytical technology (PAT). The second presenter will describe efforts aimed at establishing a design space and how the knowledge obtained can be leveraged to develop a robust control strategy. The presentations will be followed by a 30 minute panel discussion.

  • 1:30PM - 3:00PM

    Session 2C (Clinical Development): Non-Liver Targets


    Session Chair(s):

    • Representative Invited
      FDA, United States
    • James D. Thompson, PhD
      Vice President, Pharmaceutical Development
      Quark Pharmaceuticals Inc., United States

    Clinical update for nonhepatic targets. While many oligonucleotide therapeutics target the liver to exploit the propensity of hepatic delivery, a substantial number of clinical programs aim at non-hepatic targets for activity. Viral replication has always been a prime candidate for therapeutic intervention with oligonucleotide therapeutics. In fact the first antisense oligonucleotides in clinical trials and the first to be approved targeted viral replication. In this session two of the talks describe more recent efforts to use siRNA to inhibit viral replication: one with a locally delivered siRNA and the other with a systemically delivered siRNA targeting the Ebola virus.

  • 3:00PM - 3:30PM

    Refreshment Break
  • 3:30PM - 5:00PM

    Session 3A (Nonclinical): Immunogenicity Assessment for Oligonucleotides


    Session Chair(s):

    • Rosanne Seguin, PhD
      Academic Associate
      McGill University, Canada
    • Representative Invited
      FDA, United States

    Instances of immunogenicity have been shown to affect the safety and pharmacokinetics of individual drug products. Is immunogenicity an important consideration for oligonucleotide therapeutics? This session will begin with a general overview of oligonucleotide immunogenicity then continue with presentations exploring assessments of immunogenicity associated with short- and long-duration oligonucleotide therapy.

  • 3:30PM - 5:00PM

    Session 3B (CMC): Novel Excipients and Other Recent Advances in Formulation Development


    Session Chair(s):

    • René Thürmer, PhD
      Deputy Head Unit Pharmaceutical Biotechnology BfArM
      Federal Institute for Drugs and Medical Devices, Germany

    In large part, the successful development of oligonucleotide therapeutics is predicated on the development of safe and effective drug product formulations. The session will examine recent advances in formulation development, including those in the field of liposomal, polymer and nanoparticle-based delivery vehicles. Strategies aimed at mitigating or eliminating the toxicity associated with oligonucleotide formulations will be presented. Recommendations for designing studies to determine potential toxicities of novel and functional excipients will be discussed. Two presentations will be followed by a 30 minute panel discussion.

  • 3:30PM - 5:00PM

    Session 3C (Clinical Development): Liver Targets


    Session Chair(s):

    • Richard Stephen Geary, PhD
      Senior Vice President, Development
      Isis Pharmaceuticals, Inc., United States
    • Representative Invited
      FDA, United States

    This session will provide updates on antisense and siRNA development programs focused on liver targets. Included in this session are two second generation antisense drugs targeting apoC-III and C-reactive protein (CRP), respectively, as well as two approaches to delivering siRNA for targeting transthyretin in TTR amyloidosis. All of these disease associated targets are primarily synthesized in the liver. The session provides insights into clinical study designs and development plans as well as proof of concept and safety assessments for two platform approaches to targeting mRNA transcripts in the liver.

  • 5:00PM - 6:00PM

    Networking Reception

Day 2 Thursday, September 26, 2013

  • 8:30AM - 10:00AM

    Session 4 (Nonclinical, CMC and Clinical Development): Poster Session Presentations


    Session Chair(s):

    • Session Chair Invited
      United States

    This meeting offers a unique opportunity for industry and regulators to discuss the successes, challenges, and “pearls of wisdom” in oligonucleotide therapy development. This poster session presents another forum for attendees to share and discuss recent advances in the field. Posters will encompass clinical, nonclinical, CMC, and clinical pharmacology issues, and one poster from each track will be chosen for presentation during this session.

  • 10:00AM - 10:30AM

    Refreshment Break
  • 10:30AM - 12:00PM

    Session 5A (Nonclinical): Oligonucleotide Regulatory Experience


    Session Chair(s):

    • Arthur A. Levin, PhD
      Executive Vice President R & D
      miRagen Therapeutics, United States
    • Representative Invited
      FDA, United States

    Do oligonucleotide therapeutics represent a special class of drugs warranting specific regulatory policies? Is sufficient evidence available to guide new regulatory policy? This session will be devoted to examining what evidence is available to support class-specific regulation for oligonucleotides. Specifically, the contributions of structure and sequence to oligonucleotide toxicity will be discussed in complimentary presentations from Pharma and FDA representatives.

  • 10:30AM - 12:30PM

    Session 5B (CMC): Analytical Advancements and Oligonucleotide Specifications


    Session Chair(s):

    • Nigel R. Richardson
      Analytical Manager
      GlaxoSmithKline, United Kingdom

    Drug substance and drug product specifications focus on critical quality attributes useful for ensuring safety and efficacy. In this regard, the general requirements to confirm identity, determine strength and assess impurities apply to oligonucleotides. However, the size and complexity of oligonucleotides relative to small molecule drugs can make fulfilling these basic requirements challenging. A review of the state of the art of oligonucleotide analysis will be presented. The presentation will enable a discussion of the challenges associated with developing and justifying meaningful specifications for oligonucleotide therapeutics.

  • 10:30AM - 12:00PM

    Session 5C (Clinical Development): Oncology


    Session Chair(s):

    • Steve Hughes, MD
      Vice President, Clinical Development
      Isis Pharmaceuticals, Inc., United States
    • Representative Invited
      FDA, United States

    Clinical programs utilizing oligonucleotide therapeutics have been around since the days of polyIC induction of interferon as a mechanism of oncolytic activity. These programs were followed by attempts using first generation phosphorothioate oligodeoxynucleotides to target of single genes known to be critical for tumor survival or replication. This session will focus on more recent efforts to use newer generation antisense (Gen2.5) and siRNA to target key genes and a novel approach of administering a microRNA mimetic that represses key evolutionarily conserved pathways to alter tumor growth.

  • 12:00PM - 1:30PM

    Lunch
  • 1:30PM - 3:00PM

    Session 6: Pharma/FDA Case Study


    Session Chair(s):

    • David H. Schubert
      Vice President of Regulatory Affairs & Quality Assurance
      MediVector, Inc., United States

    Oligonucleotide therapeutic development presents various challenges to CMC, Pharm/Tox, Clinical Pharm., and Clinical disciplines at early-, middle-, and late-stage development. This session will include three real and hypothetical case studies that identify challenges faced by these disciplines across the development cycle. Case study presenters will engage the audience in discussions about development issues, decision-making, and lessons learned based on prior experience.

  • 3:00PM - 3:30PM

    Refreshment Break
  • 3:30PM - 5:00PM

    Session 7A and C (Nonclinical and Clinical Development): Translational Assessment of Oligonucleotide Effects in the Kidney


    Session Chair(s):

    • No-image Jan Kevin Losos, PhD
      Director, Safety Assessment Projects
      GlaxoSmithKline, United States
    • Representative Invited
      FDA, United States

    In this joint session for the nonclinical and clinical tracks, the importance of renal toxicology for oligonucleotide therapeutics will be considered. Renal toxicity occurs in a dose-related fashion for some oligonucleotide products in nonclinical studies. Are nonclinical results predictive of what can be expected with therapeutic doses in clinical administration? This session will discuss two instances of renal toxicology in nonclinical studies as well as potential underlying mechanisms including exaggerated pharmacology. A final presentation will examine an instance of renal toxicity associated with the clinical administration of an oligonucleotide and its importance.

  • 3:30PM - 5:00PM

    Session 7B (CMC): CMC Strategy and Summary of Regulatory Interactions for Mipomersen


    Session Chair(s):

    • Anthony Scozzari
      VP, Development Chemistry & Manufacturing
      Isis Pharmaceuticals, Inc., United States

    The first systemically administered antisense oligonucleotide was recently approved in the US. Various aspects of the Mipomersen chemistry, manufacturing and control (CMC) strategy, including the approaches taken for control of materials, process development and validation, analytical methods and specifications will be presented. Interactions between the sponsor and the US and European regulatory authorities on the content of the Mipomersen Common Technical Document (CTD) will be discussed. Two presentations will be followed by a 30 minute panel discussion.

Day 3 Friday, September 27, 2013

  • 8:30AM - 10:00AM

    Session 8 (Nonclinical, CMC, Clinical Development): Hot Topics


    Session Chair(s):

    • Arthur A. Levin, PhD
      Executive Vice President R & D
      miRagen Therapeutics, United States
    • Representative Invited
      FDA, United States

    Hot Topics will present cutting-edge information of interest to all three tracks. This year the hot topics will focus on miRNA roles in disease and therapy, and a novel form of crop protection against insects based on ingestion of siRNA. As a whole the presentations will explore rivaling forces governing RNA expression in maintaining, enhancing, and disrupting function.

  • 10:00AM - 10:30AM

    Refreshment Break
  • 10:30AM - 12:00PM

    Session 9: Conference Highlights


    Session Chair(s):

    • Session Chair Invited
      United States

    Session 9: Conference Highlights

  • 12:00PM - 12:15PM

    Closing Remarks

Registration Fees 

Other Fees

Charitable Nonprofit/Academia
$675.00
Government (Full Time)
$405.00
Industry
$1350.00
Group Discounts

Printable Registration Form

Register Online

CANCELLATION POLICY: All cancellations must be received in writing two weeks before
the start of the event. Administrative fee that will be withheld from refund amount:

  • Member or Nonmember = $200
  • Government or Academia or Nonprofit (Member or Nonmember) = $100
  • Tutorial (if applicable) = $50

Cancellations must be in writing and be received two weeks before the start of the event. Registrants who do not cancel two weeks before the start of the event and do not attend the event will be responsible for the full registration fee. Registrants are responsible for cancelling their own hotel and airline reservations. DIA reserves the right to alter the venue, if necessary. If an event is cancelled, DIA is not responsible for any airfare, hotel or other costs incurred by registrants.

Unless otherwise disclosed, the statements made by speakers represent their own opinions and not necessarily those of the organization they represent, or that of the Drug Information Association. Speakers, agenda and CE information are subject to change without notice. Recording of any DIA educational material in any type of media is prohibited without prior written consent from DIA.

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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