Theme 1: Preclinical Development of Novel Therapeutic Modalities and Approaches

Recent breakthrough advances in basic sciences have led to an explosion of novel therapeutic modalities and approaches beyond traditional small molecules or monoclonal antibodies to treat diseases with significant unmet medical need. Examples include non-covalent small molecule inhibitors, small molecules modifying RNA, and a plethora of approved novel modalities such as CAR-Ts and anti-sense oligonuleotides. A major breakthrough has been our ability to efficiently modulate previously inaccessible pharmacological targets (e.g., DNA). These novel approaches pose unique challenges not commonly encountered in the traditional therapeutic modality space. This theme will focus on strategies, challenges, lessons learned, and case studies in the preclinical development of these novel small molecule and biologic therapeutic approaches. Examples of topics include preclinical development (e.g., biotransformation, efficacy, safety, relevant preclinical models, translational PK/PD) for novel modalities (e.g., cell and gene therapy, siRNA, vaccines, oligonucleotides, bispecifics, ADCs, peptides, millamolecules), novel routes of delivery, design modifications to improve bioavailability and exposure at the site of action, and targeted delivery utilizing endogenous transporters/enzymes.

Theme 2: Innovation and Acceleration of Preclinical Development

Bringing transformative therapeutics to patients faster is a constant endeavor in the pharmaceutical field. Novel in vitro and in vivo models, as well as computational approaches, are making it possible to innovate and accelerate drug development as never before. This theme will focus on novel methods and approaches to optimize drug candidates for ADME, efficacy, safety, identify biomarkers, and translate findings to the clinic to enable better prediction of first-in-human dose, efficacious dose/regimen, and assessment of therapeutic index. Topics include in vitro and in vivo models for ADME, efficacy and toxicity evaluations, candidate optimization and screening strategies, biomarker development, and imaging technologies. Additionally, innovative computational approaches such as mechanistic PK/PD modeling, predictive toxicology, quantitative systems pharmacology, advanced analytics, artificial intelligence/machine learning, as well as in vitro drug development paradigms will be discussed.

Theme 3: Advances in Preclinical Development of Immuno-Therapeutics

Targeting the immune system for numerous diseases including cancer, autoimmune diseases, inflammatory diseases, neurology, and ophthalmology has tremendous breakthrough implications for patients. Immuno-therapeutics have expanded into a wide range of formats from biologics, small molecules, and novel modalities. Major advances such as development of micro-physiological systems, experimental pharmacology assays, systems immunology models, and use of “reverse translation” to guide the preclinical development of immune-therapeutics are under intense investigation. There are also several unique challenges in the preclinical development of these therapeutics including relevance of preclinical efficacy and safety models, evolving understanding of mechanism of action, translational experience for monotherapy and combinations, and regulatory considerations. This theme will focus on recent advances and learnings for various immuno-therapeutics and innovative strategies for their preclinical development. Topics include pharmacokinetics, pharmacodynamics, optimal therapeutic design, biomarkers, biodistribution, imaging, predictive in vitro, in vivo, and in silico models for efficacy and safety, assessment of therapeutic index, translational aspects for prediction of human PK, efficacious dose and regimen, combinations, and recent case studies to highlight successes, challenges, and lessons learned.

Theme 1: Will the Context of Use for Each Assay Please Stand Up?

Defining the context-of-use (COU) is essential to establish the purpose in fit-for-purpose biomarker assay validation. This theme will focus on the challenge of defining COU and the impact of COU on the entire process of assay design, development, validation and implementation. How to define COU? What to do when COU information is not available? How to continue the active dialogues between industry and regulatory agencies to shape heath authority expectations? These have been some of the hot topics in the global biomarker community, and we will continue these discussions at PharmSci 360.

Theme 2: To Boldly Go Where No Assay Has Gone Before!

Each assay has its own challenges to reach a final method that delivers the needed data. This theme focuses on the solutions—those that are stretching bioanalytical sciences in new directions. What technologies are being developed or adapted to support novel modalities? What new solutions are being applied to address current and emerging challenges?

Theme 3: Are you ready for the laboratory of the future?

The lab of the future needs to be more efficient and adaptive than ever before. What can we do to shorten critical path timelines? How are we ensuring data quality and integrity in a fast-moving, time-sensitive environment? How will we continue to do so with novel technologies being implemented for more complicated molecules?

Register for PharmSci 360 today!

Did you miss the 2020 PharmSci 360 Call for Symposia deadline? We are still accepting Rapid Fire submissions!

What are Rapid Fires?

Rapid Fires are quick, 10-min presentations with 3-min of Q&A on the latest scientific topics. Have research or ideas that are novel and exciting? Share them on the Rapid Fire stage!

AAPS is still accepting Rapid Fire programming ideas for the 2020 PharmSci 360. Submit your presentation idea by June 17, 2020, to be considered. Visit the 2020 PharmSci 360 submission instruction page to learn more about this year’s programming—including the tracks, topics, and themes it will cover—as well as how to submit your proposal.