PharmSci 360

Theme 1: Out-of-the-Box Approaches for Innovation

Keywords: Multi-Targeted Therapies, Microbiome-Based Therapeutics, Immune Modulation Therapy, Novel Drug-Delivery Systems, Combination Therapies, Targeted Therapeutics, GPL-1 Agonist, Gene and Cell Therapies, Regenerative Medicine, Epigenetics, Cancer, Infectious Diseases, Autoimmune Diseases, Allergies, Metabolic Diseases, Neurodegenerative Disorders, Inflammation, Rare Diseases

This theme spotlights groundbreaking strategies that address unmet medical needs. Topics include multi-targeted therapies, personalized precision medicines, and next-generation immune modulation strategies for difficult-to-treat diseases such as cancer, metabolic diseases, autoimmune conditions, allergies, and inflammation. Focus areas may include mechanistic insights and therapeutic potential of microbiome-based interventions, innovative delivery systems for hard-to-reach targets, expanding the applications of GLP-1 receptor agonists in and beyond metabolic and cardiovascular diseases, combination therapies, exosome-based therapies, cell and gene therapies, regenerative medicine, immunotherapies, and immune-engineering or immune modulation strategies. Proposals should highlight interdisciplinary approaches to therapeutic design that enable access to difficult targets and combining therapies for interdependent effects. 

Theme 2: Emerging Technology as a Game Changer in Drug Discovery

Keywords: AI and Advanced Robotics, Prediction, Digital Tools, Imaging, High-Throughput Screening, CRISPR, Precision Medicines, Targeted Therapeutics, Single-Cell Techniques, Drug Delivery, Nanotechnology, Biopharmaceutics, Advanced Biomaterials, Cell-Materials Interaction, Biomimetic models, Organs-on-Chips, RNA Vaccines Engineering

We look to examine cutting-edge, enabling advancements in drug discovery and development and showcase emerging technologies designed to overcome key development bottlenecks, specifically: 

• High-throughput screening enhanced by imaging-based or CRISPR-based selection
• Latest advancements in sequencing, protein engineering, and tissue engineering for precision medicine.
• New biomimetic models, such as organoids and organs-on-chips, as foundational validation systems
• Direct imaging for in-depth mechanistic insights into cell-material interactions, single-cell sequencing to address heterogeneous cell responses
• High-resolution structural biology for elucidating protein-drug interactions

Theme 3: How can we Advance Basic Research as the Bedrock of Pharmaceutical Science?

Keywords: CRISPR-Based Therapeutics, mRNA vaccine, Antibiotics for Drug Resistant Microorganisms, Cell-Based-Therapeutics, Antibody-Drug, Biomarkers, Amyloid fibrils, Blood-Brain Barrier, Membrane Permeation, novel modalities, basic disease research, unmet medical needs, novel modalities

This theme is looking to explore how to better connect fundamental and mechanistic disease research at the pathophysiological and molecular levels with early industry-sponsored drug discovery. How can industry/academic partnerships better leverage diverse expertise and resources to accelerate innovation? Case studies that explore this space are welcome.

Theme 1: Novel In Silico and In Vitro Approaches in Drug Development

Keywords: Animal models, Imaging, AI/ML Incorporated Development, In Silico Models, PK/PD Modeling, In Vitro Assays, Organ-on-Chip Systems

The growing complexity of new modalities and expanded options for rare diseases through cell and gene therapy applications has driven translational science’s need for improved models to conduct proof-of-concept studies. Likewise, the growing effort to reduce and refine animal use has rapidly accelerated improvements to in silico models and in vitro test systems, e.g., organ-on-a-chip systems, necessitating updates to current regulatory frameworks. This track focuses on in vitro systems and tools that provide key translational insights, novel in silico modeling and simulation approaches, and physiologically based PK/PD modeling. Proposals should highlight the translational potential of the work that either is immediately applicable to accelerated drug discovery and development or serves to advance updated regulatory frameworks and guidance. 

Theme 2: Designing and Delivering Effective Clinical Trials for Specific Populations 

Keywords: Enrollment, Data Integrity, Continuous Monitoring, Real World Evidence, Disease-state Simulations, PK, Pediatric, Geriatric, AI/ML 

This theme explores the current challenges, solutions, and new innovations associated with clinical trial design and implementation. Topics may include trial considerations for specific disease populations and specific populations (e.g. pediatrics, pregnant individuals, the elderly); real-world evidence; disease-state simulations; PK modeling and results for specific populations; clinical trial design innovations; practical examples of innovation in clinical operations; population considerations; in-home devices and personal e-data collection; and the use of machine learning and AI to inform clinical trial enrollment, design, and/or outcomes.

Theme 3: Translating Animal PK, Toxicology, and Biomarkers to Clinical Success

Keywords: Model-Informed Drug Development, Clinical Outcome Assessments, PK/PD Endpoints, Composite Endpoints, Digital Health and Real-World Data, Imaging Endpoints, Animal Data, Animal Models Biomarkers

How do we better establish the relevance of PK/PD and/or biomarker assessments from preclinical species to clinical settings? We explore the use of biomarkers in diverse trial functions, the application of surrogate endpoints to clinical trials, the connection of animal biomarkers, with outcomes to human outcomes, and PD-driven trials. Case studies including the following are desired: clinical outcome assessments (patient, observer, and clinician reported outcomes), composite endpoints, digital health and real-world data (wearables and sensors, electronic records, mobile health apps and telemedicine); PK/PD endpoints; and imaging endpoints (MRI, CT, PET scans as well as echocardiography, optical coherence tomography, etc.). Other case study submissions can examine the use of animal data to support downstream clinical biomarker use and design. Biomarker-related submissions should elucidate how the interpretation of the biomarker data is applied to preclinical or clinical settings. Submissions focused on method development/validation or technical design of biomarker assays should be directed to the Bioanalytics Track.

Theme 1: Regulatory Guidance for AI/ML - Quo Vadis?

Keywords: ICH M10, Method Development, Method Validation, HRMS, Regulated Environment, AI, Machine Learning, ChatGPT, Modeling, MHRA, EMEA, FDA, CFR, 510K, PMA, IVDR, Hybrid Assays, High-Resolution Mass Spectrometry, Predication, Documentation.

How can industry collaborate with regulatory agencies to update bioanalysis regulatory framework for AI and ML? Proposals should explore industry/regulatory collaboration regarding data, evolving regulations, and enhanced transparency and documentation; case studies are also welcome. How can innovation and flexibility in regulatory approaches balance the need for harmonization and predictability in submissions? 

Theme 2: How Bioanalysis and Translational Science Accelerate Drug Development

Keywords: Biomarker, PK/PD, EMEA, MHRA, FDA, 510K, PMA, IVDR Devices, Pharmacokinetics, Pharmacodynamics, Efficacy, Liquid Biopsy, Personalized Medicine, Regulatory Expectations, Flow, Exosomes, cDNA, ctDNA, Genomics, Mass Spectrometry, LC/MS, Quantitation, Patient-Centric Applications, Sampling, RRR, Clinical, Preclinical

This theme explores the use of biomarkers for pharmacodynamic purposes, highlighting innovative approaches and best practices. We seek proposals with a focus on liquid biopsy technologies, disease-monitoring, safety, efficacy and enrollment biomarkers, as well as those used as surrogate and primary endpoints, and adherence to regulatory expectations. Sessions should advance the field of PK/PD relations and promote the development of more effective and personalized therapeutic strategies. Presentations that examine techniques and methodologies for quantitatively evaluating biomarkers indicative of therapeutic response/advances; applications of liquid biopsy to address biomarker detection/monitoring/quantitation; best practices for biomarker measurement integration; opportunities for technical and scientific discussions between the scientific community and regulators; and compliance of biomarker measurement in line with the various regulatory expectations are encouraged. 

Theme 3: Cutting-Edge Bioanalysis Instrumentation and Techniques for Novel Therapeutic Modalities--

Keywords: ADC, ddPCR, PCR, Peptide, LC-MS, HRMS, PROTAC, Exosomes, Immunogenicity, NAb, Patient-centric Sampling Technology, Novel, Nextgen Sequencing, Microfluidics, Biosensor, Sensitivity, Specificity, Novel Modalities

This theme examines cutting-edge bioanalytical techniques and methodologies for analyzing novel therapeutic modalities. It emphasizes the specific challenges and solutions associated with novel modalities such as antibody-drug conjugates (ADCs), gene and cell therapies, and peptide-based treatments. Submissions exploring the latest advancements in bioanalytical platforms, such as PCR, high-resolution mass spectrometry, next-generation sequencing, flow cytometry and spectroscopy, and bioanalytical microfluidics, are welcome. This theme explores the impact of these novel platforms on sensitivity, specificity, throughput, and data quality in bioanalysis. It also discusses how integrating these advanced techniques into existing workflows can enhance analytical capabilities and efficiency.

Theme 1: Advancements in Manufacturing and Characterization of Pharmaceuticals/ Biopharmaceuticals

Keywords: Continuous Manufacturing, Flow Chemistry, Single-Use Technologies, Modality Agnostic Flex Facilities, Green Chemistry, Sustainability, Sterile Manufacturing, Combination Drug Product, Container Closure, Packaging, Robotics, Smart Manufacturing, Bioprocessing Innovation, Pharmaceutical Manufacturing 4.0, point-of-care manufacturing, flexible facilities, Life Cycle Management, EU Regulatory Reform, ICH Q12 And Q14, Chromatography, PAT, AI/ML For Manufacturing

This theme explores innovations in manufacturing and characterization of small molecules and biological products and APIs. This could entail a single novel technology, a combination of methods that incorporate novel technologies, or the innovative application of established techniques for drug manufacturing and characterization. Regulatory considerations for implementing advanced manufacturing, lifecycle management strategies for advanced manufacturing, and topics related to FDA guidance on “Advanced Manufacturing Technologies Designation Program” are also covered.

Theme 2: Navigating the Complexities of Novel Modalities and Delivery Systems

Keywords: Manufacturing and Analysis of New Modalities, ADCs, Adjuvants, Vaccines, Oral Peptide, RNA-based Therapies, mRNA, SiRNA, Antisense Oligonucleotides, Gene Therapy, Phage Therapy, CRISPER, Bispecifics, Bioenhancement, Enabling Analytical Technologies, Analytical QbD Approaches, Biosimilar and Generic Drug Products, Novel Adjuvants, Impurity Characterization, Nitrosamines, Particulates, Nano-medicine, 3D Printing, Long-acting Injectables, Manufacturing Scalability, Long Term Sustainability

The process development and commercialization of novel modalities present significant manufacturing and quality analysis challenges. Key late-stage issues include scale-up, formulation robustness, and managing costs due to limited experience. Effective phase-appropriate strategies involve risk management practices like Quality by Design (QbD) to identify and control critical quality attributes. Case studies on navigating regulatory frameworks for new chemical entities (NCEs), biosimilars, and generic drug products that require applying agency guidelines and proactive engagement are encouraged, as are proposals that discuss analytics supported impurities management approaches. Proposals that discuss characterizing bioconjugates and quantifying impurities in mixed modality products are of special interest, as are case studies that focus on integrated quality control systems and fostering collaboration across disciplines, as these can facilitate the successful development of innovative therapeutic products.

Theme 3: Predictive Tools and Applications in Manufacturing and Product Quality 

Keywords: Mechanistic Understanding, Materials Science Tetrahedron Based Product Development, Predictive Tools, AI, Process Models, Machine Learning, Advanced Process Controls, Stability Modeling, Control Strategy, Regulatory Filing, cQAs, Multivariate Modeling, PAT, Smart Experiments, Systems Modeling, Digital Twins

This theme covers the application of predictive in silico approaches across the entire product development and manufacturing space, from materials science to GMP manufacturing and operations. This specifically includes Materials Science Tetrahedron-based approaches in product development; molecular modeling crystal structure prediction; mechanistic and data driven approaches to process development, and manufacturing, as well as material sparing predictive experimental approaches; use of individual unit operations modeling digital twins; systems modeling; PAT and/or smart experiments in product characterization, scale-up, manufacturing failure modes and risk assessment. Other areas of focus include novel approaches in control strategy and regulatory filing; predictive stability applications in determining analytical critical quality attributes (CQAs); enhanced stability modeling; data analysis to characterize variability in GMP manufacturing processes; and other relevant predictive tools and applications. App

Theme 1: Accelerating Drug Development Through Digital Innovation

Keywords: In Silico Modeling, AI, Machine Learning, High-Throughput Screening, Computational Optimization, Drug Stability Prediction, Tissue Targeting Design, Regulatory Considerations

In silico modeling, machine learning, and AI are revolutionizing pharmaceutical development by providing precise, data-driven solutions to accelerate drug discovery, formulation, and delivery. This theme focuses on the application of AI/machine learning approaches and other digital innovations in key areas of drug development such as excipient selection, drug stability prediction, formulation development, and manufacturing process optimization. Computational modeling plays a crucial role in optimizing drug absorption and its impact on pharmacokinetics, enhancing the predictability of clinical outcomes. Advances in digital modeling also facilitate tissue-targeting design, improving drug delivery specificity, and therapeutic efficacy. Furthermore, the evolving regulatory landscape for AI-driven drug development is addressed, including considerations for model validation, data integrity, and regulatory acceptance of computational approaches. Case studies highlighting the above aspects are particularly encourage

Theme 2: Enabling Technologies and Materials for Formulation Innovation 

Keywords: Advanced Materials, Targeted Delivery, Lipid Nanoparticles, Polymeric Nanoparticles, RNA Therapeutics, Biologics Stabilization, Controlled Release, Surfactant, Gene Delivery, Impurity Profiling, Stability and Shelf Life, Regulatory Considerations

The increasing complexity of modern therapeutics requires new formulation technologies and novel materials to enhance drug delivery, stability, and manufacturing. This theme focuses on key enabling technologies and materials that drive innovation in formulation science including lipid-based and polymeric systems, biologics stabilization, and strategies to optimize solubility, bioavailability, and controlled release. Additionally, discussions cover the stabilization of RNA therapeutics and gene-editing modalities, along with impurity profiling strategies and their impact on formulation stability and shelf life. Regulatory considerations should also be addressed, including pathways for approving novel formulation technologies and how to ensure compliance with evolving regulatory guidelines. 

Theme 3: Patient-Centric Formulation and Personalized Drug Delivery Strategies

Keywords: Patient-Centricity, Drug-Device Combinations, Non-Invasive Delivery, User-Friendly Formulations, High-concentration Biologics, Subcutaneous Delivery, Device Development, Primary Container, Drug-Device Interaction, Personalized Therapies.

Patient-centric drug development is a key driver of pharmaceutical innovation, ensuring therapies are convenient, accessible, and aligned with patient needs. This theme examines new drug formulation and delivery strategies that improve patient experience and compliance. Topics include subcutaneous delivery of high-concentration biologics, drug-device combinations like wearable injectors and closed system drug-transfer devices, and non-invasive administration routes such as pulmonary, intranasal, and intradermal delivery systems. Discussions should focus on overcoming technical challenges in patient-centric formulations, enhancing patient experience through sustained-release products and long-acting injectables, and leveraging quality target product profiles (QTPPs) to streamline product development. Other topics for consideration are customization of formulations for personalized therapies, including tailored excipient selection and formulation strategies for pediatrics, geriatrics, and special populations;

Today’s pharmaceutical scientists must manage their own professional development and advancement in an increasingly complex and dynamic environment. This includes proactively determining the right skills to develop, which scientific areas to pursue, and what strategies to employ as they navigate the job market.

The PharmSci 360 Professional Advancement Track will present a dozen sessions that help scientists at all career stages determine where they want to take their career next and develop the skills and strategy necessary to achieve their goals. 

Attention: AAPS is especially interested in speakers who work in the pharmaceutical industry as recruiters, career coaches, talent managers, economists, financiers, and similar roles that give the speaker experience with the scientific labor market and the forces affecting today’s scientists as they develop their careers. 

AAPS is also especially interested in session proposals featuring speakers who have not participated in the PharmSci 360 Career Development track in the recent past, or who have done so with extraordinary success.

Theme 1: Building a Skillset for the Future 

Keywords: AI, Resumes, Cover Letters, Recommendations, Certifications, Linkedin, Recruiters, Interviewing, Executive Presence, Presenting

Practical job-seeking skills for early-to-mid career scientists as well as insights into how technology is changing the way hiring works. Ideal topics include choosing an employer; AI in hiring processes; developing a competitive resume; interview strategies; understanding your professional and scientific positioning as a candidate; executive presence; getting published; and presenting your science. 

Theme 2: Navigating the Business of Science

Keywords: Private Equity, Venture Capital, Investors, Networking, Interdisciplinary Science, Employment Contracts, Intellectual Property, Business Plans, Scaling, Executive Management

These sessions highlight business skills and strategies for scientists at all career stages, including identifying the scientific fields that are most promising based on your scientific position, forecasting the future of therapeutic areas for your career; employment contracts; pitching a start-up; managing and owning intellectual property; developing private and public funding; networking with key opinion leaders; and moving into the C-suite.

Theme 3: Career Strategy in an Evolving Pharmaceutical Landscape

Keywords: Change Management, Talent Management, International Relations, Business Ethics, Markets

These sessions provide enterprise-level insights into the pharmaceutical industry labor market in the U.S., and skills for planning and pursuing mid-career goals. Topics include economic and market forecasts for pharmaceutical science; how to recruit and retain key talent; managing a modern workforce; managing consultants; communicating with partners; and creating ethical workplaces.