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.
Oligonucleotide Therapeutics Society (OTS) is an open, nonprofit forum to foster academia and industry-based research and development of oligonucleotide therapeutics. Formed in 2004, OTS is a registered 501(c)6 nonprofit with the US government.
American Association of Pharmaceutical Scientists (AAPS)
The American Association of Pharmaceutical Scientists provides a dynamic international forum for the exchange of knowledge among scientists to enhance their contributions to health. They offer timely scientific programs, ongoing education, opportunities for networking, and professional development.
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. The nonclinical track sessions are 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
- 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:
- Clinical Pharmacology
- Clinical Research
- Chemistry, Manufacturing and Control
- Clinical, Regulatory, and Business Development
- Delivery Technologies
- Drug Discovery
- Quality Assurance
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.