Tracks and Themes
Track 1: Inspiring Innovation in Formulation, Bioprocessing and Drug Delivery
Keywords: Formulation, Drug Delivery, Bioprocessing and manufacturing, Process Analytical Technology (PAT), Machine Learning (ML), Precision Medicine, Patient-centric approaches, Devices, Quality Control, Scale-up
This track focuses on the latest advances and challenges in formulation and delivery of biologics for newer and established modalities, including multi-specific antibodies, antibody-drug conjugate (ADCs), peptides, vaccines, nanomedicines, cell and gene therapies, and how prior knowledge along with current approaches can accelerate their development and delivery. It will delve into how novel technologies can improve the bioprocessing, characterization, delivery, and comparability of biopharmaceutical products, as well as the efficiency and quality of production through continuous processing, and process analytical technology (PAT) methodologies. Furthermore, the track will highlight the advances in patient-centric technologies, such as point-of-care and diagnostic testing, devices, and personalized medicine, and how they can offer novel and tailored therapies for various indications. The track encourages the integration of cutting-edge technologies like AI and machine learning (ML) to improve formulation, bioprocessing, quality, and personalization of biologic therapies and ultimately enhance patient outcomes and compliance.
Theme 1: Accelerating Novel Formulation Development of Biologics for Newer and Established Modalities
Keywords: Formulation Development, Novel Delivery Systems, High Concentration Formulations, Oral Delivery, Stability, AI, Design of Experiments (DOE), Lipid Nanocarriers, 3D-Printed Delivery Systems
Theme 1 will explore the latest advances in formulation and delivery of complex biologics via novel dosage forms, lipid nanocarriers, 3D-printed systems, and targeted RNA approaches in addition to traditional modalities. The development and delivery challenges included in the scope relate to the cold-chain storage, transportation, injectability of high viscosity/concentration formulations, oral administration of biologics, successful implementation of novel technologies in drug delivery and controlled-extended dosage forms with improving the stability, bioavailability, and patient compliance. The theme will also focus on the application of AI, prior knowledge on accelerating the high throughput screening, design of experiments, and formulation technologies to expedite product development.
Theme 2: Bioprocessing, Characterization & Comparability for Complex Biologics
Keywords: Bioprocessing Optimization, Media Optimization, Clone Selection, Characterization, AI, DOE, CAR T, Cell Therapy, Immunotherapy, Potency Evaluation Assays, Continuous Bioprocessing, Product Quality Monitoring
Recent advancements in bioprocessing involve using complete living cells or their components to optimize both upstream and downstream processes. This includes addressing host cell complexities, improving their expression, and ensuring the characterization and comparability of biopharmaceutical products. These steps are essential for developing safe and effective biologics, such as emerging RNA-based treatments. We also seek proposals in the areas of structural biology, protein engineering of novel antibody formats for enhanced tissue penetration and target engagement, and strategies for improving successful outcomes for biotherapies such as immunotherapies and CAR T cell therapies. This theme will explore pathways to enhance bioprocessing processes, including media optimization, clone selection and most importantly predicting process parameters through the implementation of computation models, AI, ML and smart DoEs. The theme will also showcase the latest advances in continuous bioprocessing, real-time product quality monitoring, PAT, and ML applications in biopharmaceutical production.
Theme 3: Patient-Centric Product Design and Precision Medicine
Keywords: Patient-Centric Design, Point of Care, Diagnostic Testing, Devices, Personalized Medicine, Genomics, Proteomics, CRISPR, Microbiome, Living Medicines
Theme 3 explores several facets of Precision Medicine that intersect to improve patient outcomes. Advances in patient-centric technologies and how point-of-care and diagnostic testing can increase the therapeutic effectiveness and compliance of patients, including improved drug delivery using combination devices, ultimately improving the product adherence and clinical outcomes. Personalized medicine, such as genomic and proteomic approaches, CRISPR-based gene editing, microbiome manipulation, and living medicines would be explored for developing novel and tailored therapies for various diseases and conditions. The theme will also address the applications of AI and ML in clinical testing, product labelling, and data review to further precision medicine.
Track 2: From Bench to Bedside: Bioanalysis and Translational Science
Keywords: ADME (Absorption, Distribution, Metabolism, and Excretion), Antibody-Drug-Conjugates (ADC), AI (Artificial Intelligence), Assay development, Novel Bioanalysis, Biomarkers, CAR-T, Clinical Translation, Gene Editing, Gene/Cell Therapies, Global Trials, in vitro assays, LDT, Lipid nanoparticles (LNP), patient-centric sampling, Pharmacokinetic-Pharmacodynamic (PK/PD) Correlations, Protein Degraders, Regulatory CLIA, IVD, IVDR, Safety, Sample management, RNA Therapeutics
The rapid rise of newer (now beyond novel) therapeutic modalities has warranted innovative approaches and strategies to analyze these newer drug products and their respective constituents. Newer modalities, including but not limited to CAR-T cells, gene therapies, and protein-based drugs, demand innovative approaches to assess their pharmacokinetics (PK), pharmacodynamics (PD), and safety. Furthermore, identifying and validating relevant biomarkers to predict efficacy and monitor toxicity is essential but often challenging due to the unique mechanisms of action of these modalities. Addressing these complexities requires a multidisciplinary approach and cutting-edge analytical techniques to support clinical development and patient care. Additionally, there is a widespread industry interest in evaluating the predictive accuracy of preclinical ADME/PK/PD and clinical pharmacology data, as well as Quantitative Systems Pharmacology (QSP) modeling, in translating to clinical outcomes. This track focuses on innovative bioanalytical techniques and translational tools including in-vitro and in-silico modeling to predict clinical outcomes. This track focuses on innovative bioanalytical techniques and translational tools including in-vitro and in-silico modeling to predict clinical outcomes.
Submissions are invited on topics related to innovative bioanalytical approaches for newer modalities, including protein degraders, oligonucleotides, siRNA, LNP-mRNA/DNA, AAVs, cell therapies, peptides, antibody-drug conjugates (ADCs), and other biologics. In addition, we seek presentations that demonstrate how bioanalytical data (PK, immunogenicity, PD and target engagement), in combination with other translational information, can inform optimal human dose projections and phase 2 regimen design through predictive PK/PD modeling.
Theme 1: Breaking Barriers: Bioanalytical Innovation for Complex Biologics
Keywords: Assay Sensitivity, Anti-Drug Antibodies, Clinical Translation, Assay Development; Novel Bioanalytics, Clinical Data Sensitivity, Immunogenicity
Bioanalytical methods may not be sensitive enough to generate translatable pharmacokinetic, pharmacodynamic, and target engagement data to support clinical translation. This theme explores bioanalytical space surrounding newer modalities, with a focus on lipid nanoparticles (LNP), nanoliposomes, mRNA, siRNA, protein degraders, and gene therapeutics. Additionally, factors such as large molecular weight, heterogeneity, soluble target engagement, natural ligands, and potential immunogenicity complicate the development of sensitive and specific assays. An industry-wide challenge is understanding whether higher titers of anti-drug antibodies from highly sensitive assays correlate to a decrease in drug product efficacy. This Bioanalytical theme also aims to discuss data sensitivity, such as ADA/Nab assays, and its real impact on clinical data. We would like to welcome submissions that share experiences and data for these topics.
Theme 2: Managing Global Clinical Trials and Crises in Sample Logistics
Keywords: Global Trials, Inclusion Criteria, Exclusion Criteria, Patient Sampling, In Vitro Diagnostic Medical Devices Regulation, IVDR, Clinical Laboratory Improvement Amendments, CLIA, Regulatory Concerns, Sample Management, Artificial Intelligence, Machine Learning, Recruitment, Trial Design, Recruitment.
Global clinical trials have expanded in scope and diversity, but managing patient samples has become increasingly complex. Regulations, such as the U.S. CLIA and the recent EU IVDR, affect clinical sample analysis, posing logistical challenges. AI aids in trial design, patient recruitment, and sample management, though AI can introduce bias. This theme invites submissions to share experiences in managing global trials, utilizing AI, addressing regulatory concerns, and ensuring compliance with evolving guidelines to streamline patient sampling and analysis processes in global clinical trials. Additionally, insights on managing overlaps and differences in bioanalytical regulations, and practical solutions to common logistical hurdles in global clinical trials, are encouraged.
Theme 3: Preclinical and Clinical PK/PD, Safety Translation of Newer Modalities
Keywords: In Vitro Assays, Biomarkers, Absorption, Distribution, Metabolism and Excretion, ADME, Pharmacokinetics and Pharmacodynamics, PK/PD, Clinical Pharmacology, Clinical Translation, Predictive Safety, Protein Degraders, Oligonucleotides, siRNA, Gene/Cell therapies, Peptides, Antibody Drug Conjugates, Organ-on-a-chip, Gene Editing
Traditional in vitro assays often have limited predictive abilities for newer modalities, and the bioanalysis of these newer modalities is often complex and multilayered, with challenging sample storage and preparation. As newer therapeutic modalities advance through preclinical and clinical development, many lead drug candidates have entered clinical trials, with several achieving coveted regulatory approvals. This theme will explore ADME, PK/PD, clinical pharmacology, biomarkers, safety, and additional challenges in developing newer modalities such as protein degraders, oligonucleotides, siRNA, LNP-mRNA/DNA, AAVs, gene/cell therapies, peptides, and ADCs. It will also explore the use of in vitro tools to correlate PK-PD, such as MPS and organ-on-a-chip systems. Submissions are encouraged to share experiences, both successful and unsuccessful, in translating preclinical research into clinical development. This includes insights on overcoming specific challenges, optimizing PK and PD profiles, managing safety concerns, and identifying reliable biomarkers for these novel therapies. Discussions on regulatory hurdles, innovative approaches to ADME characterization, and strategies for improving clinical pharmacology and patient outcomes are also welcome.