What's Next in Precision Medicine? Small Molecule Biomarkers to Personalize Disease Prediction, Diagnostics and Therapies

Tuesday, September 20, 2022,12:30-2:00pm ET

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While next-generation sequencing has been instrumental in advancing genomics-based precision medicine, the full potential of personalized therapy has yet to be realized at scale. DNA biomarkers help identify genetic variants associated with disease susceptibility and therapeutic response, but are largely static measures of heritability. This has driven exploration further down the “omics cascade” to identify more dynamic indicators of biological processes, disease progression, and treatment responsiveness. Metabolomics, or the measure of circulating small molecule biomarkers, can fill the gaps in understanding of genotype-phenotype relationships by providing real-time views of biological processes occurring across systems and eludicating how non-genetic factors interact with genetics and contribute to disease.

In this session, we will discuss how the mapping of small molecule biomarkers in blood delivers complementary information relative to traditional DNA and protein biomarkers, which can be used in drug development programs to predict disease risk, identify patient subgroups, and create more specific, efficacious therapies aligned with a patient’s unique disease biology.

Speaker: Mohit Jain, MD, Ph.D., Sapient

Enabling Patient-Centric Nanoparticle Drug Discovery and Design through Sparse-Data AI

Tuesday, September 27, 2022 12:30-2:00pm ET

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This presentation will run through a range of solid form strategies for increasing oral bioavailability and their relative advantages and disadvantages, including salt formation, co-crystallization, spray drying of amorphous solid dispersions, and Controlled Expansion of Supercritical Solutions (CESS®). The dissolution behavior of nanoparticles created using different techniques will be explored, along with the importance of crystallinity in the context of drug design.

To this end, the presentation will feature case study examples, including piroxicam (PRX), fenofibrate (FEN) and budesonide (BUD), demonstrating methods for controlling crystallinity, particle size and polymorphism using supercritical carbon dioxide to achieve the desired particle attributes.

A closer look will be taken at how the use of nanoparticles can contribute to patient comfort and quality of life, examining how reducing particle size can lead to, for example, increased drug loading and lower pill burden. Finally, the presentation will also explore how sparse-data AI can be used to predict both a drug candidate’s propensity to crystallize and its amenability for a specific nanoparticle engineering technique, highlighting that through this technology it is possible to determine that 87% of all API molecules whose structure has been publicly disclosed are amenable to CESS®. Taken together, the presentation will serve to spotlight the game-changing nature of patient-centric nanoparticle drug discovery and design powered by sparse-data AI.


Christopher Worrall, Ph.D., Nanoform

Elisabetta Micelotta, Nanoform


General Formulation Considerations in Bioequivalence Assessment for Generic Drug Approval

Thursday, September 22, 2022 – 12:30-2:00pm ET

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A generic drug product and its reference listed product (RLD) can differ in excipients, formulation design and manufacture process. The design of a formulation and use of certain excipients may have a significant impact on the efficacy and/or safety of the drug product, thus making the test product not bioequivalent (BE) to the RLD or reference standard (RS). This presentation will use case studies to demonstrate the role of formulation design in BE recommendations and during the BE assessment for ANDA applications. This information will help the generic drug applicants to understand the factors that should be considered during product development and ensure that the test product is bioequivalent to the RLD or reference standard (RS).

Speaker: Hongling Zhang, Ph.D., U.S. Food and Drug Administration

2022 Draft ICH Drug Interaction Studies Global Guideline: In Vitro Highlights and Comparison

Thursday, September 29, 2022,12:30-2:00pm ET

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In June 2022, the ICH released the first draft of its harmonized Drug Interaction Studies Guideline (M12). The guideline is the result of several meetings of the Expert Working Group since 2018 with the goal of harmonizing member regulatory agencies’ guidelines to create a single guideline that will be used across all member countries. After a review period, the guideline will be adopted in early 2024.

Drug-drug interactions (DDI) may occur when taking two (or more) drugs together results in altered efficacy or behavior of one or both drugs, potentially causing adverse effects. As drug developers shepherd their drug through the development pipeline, evaluating perpetrator and victim potential is a critical component of safety testing. Generally, these studies are guided by recommendations published by regulatory authorities, which underline the utility of in vitro experiments to predict in vivo observations, justify decisions, and inform clinical study design.

Join us to hear DDI expert, Dr. Brian Ogilvie, discuss the differences between current in vitro DDI guidance from the relevant US FDA, EMA and PMDA guidance documents.

Speaker, Brian Ogilvie, Ph.D., SEKISUI XenoTech

Novel Ionizable Lipid Library for Enabling Non-Viral Delivery

Tuesday, October 11, 2022, 12:30-2:00pm ET

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The success of the mRNA-LNP COVID-19 vaccines have clinically proven the modality of lipid-based nanoparticles for genomic medicines with ionizable amino lipids being a major constituent. Effective ionizable lipids are needed for various applications that demonstrate an excellent safety profile, can effectively encapsulate nucleic acids, and can release the nucleic acids from the early endosomes of the target cell. 

The physico-chemical profiles of different ionizable lipids will depend on application, therefore analytical evaluation is essential to validating the LNP potency and performance at each stage of drug development depending on the TPP (Target Product Profile). Precision NanoSystems offers a Genomic Medicine Toolkit to enable access to microfluidic technologies, well characterized lipid libraries, and scientific expertise in formulation and process development to design and optimize vehicles for nucleic acid therapeutics, thereby providing an end-to-end solution enabling future transformative genomic medicines.

This webinar describes novel ionizable lipids used as a functional excipient for designing vehicles for nucleic acid therapeutics, advancing targeted delivery where treatment options are absent.

Speaker: Anitha Thomas, Ph.D., Precision NanoSystems

Immunogenicity Risk Assessment and Integrated Summary of Immunogenicity

Tuesday, December 6, 2022, 12:30-2:00pm ET

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Biotherapeutics have the potential to elicit undesired immune responses, i.e., immunogenicity. For more traditional biological modalities, including antibodies, proteins, and peptides, immunogenicity is often referred to anti-drug antibodies (ADA). ADA can negatively impact the PK, efficacy, and safety of biotherapeutics. Therefore, an immunogenicity assessment is performed during stages of the drug development process, utilized for IND/CTA submission and as part of the Integrated Summary of Immunogenicity (ISI) for regulatory submissions (BLA/MAA).

The special issue in the AAPS Journal, Compendium of Immunogenicity Risk Assessments: an Industry Guidance Built on Experience and Published Work, consists of 5 manuscripts from industrial experts. Each manuscript discusses risk assessment and bioanalytical strategy using a hypothetical molecule as case study. These case studies cover diverse molecule structures and risk levels, including a monoclonal antibody, a fusion protein, a bispecific antibody, a human cytokine analogue, a PEGylated enzyme, and a PEGylated growth factor. This webinar is a continuation of such efforts. The expert speakers will discuss the challenges and strategies regarding immunogenicity risk assessment, bioanalysis, and Integrated Summary of Immunogenicity and potential/proposed solutions based on their real word experience. 

Susan Richards, Ph.D., Sanofi
Joleen White, Ph.D., Bill & Melinda Gates Medical Research Institute (Gates MRI)

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