Standards in Nanotechnology for Medical Products

October 3, 2023, 12:30-2:00 pm ET

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Liposomal drug products have been approved for clinical use, with one third of the drug product submissions containing nanomaterials regulated by the FDA constituting liposomal compositions. There is a gradual trend in increasing generic product drug submissions containing liposomes with the emergence of new products containing lipid nanoparticle formulations for vaccines and therapeutic use. FDA published liposomal guidance and product specific guidances to help industry and to facilitate product review. The critical quality attributes (CQAs) to consider for these nanomaterials include size and shape heterogeneity, chemical composition, purity of the ingredients, and the surface properties, along with drug release and various stability parameters associated with these novel products. A slight variation in the CQAs of nanomaterials may lead to differential recognition by the immune system, pharmacokinetics of the drug, which may lead to an altered biodistribution, efficacy, and safety. Consensus standards through stakeholder involvement and collaboration can assist product development, quality control and facilitate the review of submission to FDA.

The purpose of this presentation is to describe the standards development process from identifying which standards need development that are most useful for industry and regulators, through the standards development process including conducting interlaboratory studies for precision and bias in the measurements. Recent progress in the development of liposome standards, approved standards, and the database of recognized consensus standards at FDA will be presented.

Speaker: Anil Patel, Ph.D., U.S. Food and Drug Administration (FDA)

Titanium Dioxide Free Drug Products: Challenges During Drug Product Development

October 5, 2023, 12:30-2:00 pm ET

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The European Medicines Agency is evaluating a ban on TiO2 in pharmaceutical products by 2024 due to genotoxicity concerns about nanoparticles which are present in grades not relevant to pharmaceutical uses. TiO2-based film coats have been around for a long time. And therefore, pharmaceutical companies have understood the risks and limitations of the various film-coats and improved upon them. TiO2 present in these film-coats provide uniform elegant appearance and serves as an effective opacifier to help mitigate the photostability risk of a drug substance where there is a liability for it. All the benefits can be achieved by using minimum weight gain that helps with processibility too. However, film coats with an alternative opacifier, such as CaCO3, rice starch, and MgCO3, may pose risks for drug product development because of lack of experience, effectiveness as an opacifier, and potential for the basic pH microenvironment leading to chemical stability risk. As the industry is gaining experience, there is a realization that the alternative opacifiers need a much higher weight gain to achieve a uniform film coat and to act as an effective opacifier but the appearance is still not as good as the TiO2 film coat. The rice starch-based film coat does not work as a good opacifier to help with photostability risk. CaCO3 works as a relatively better opacifier but having a basic pH microenvironment creates a stability risk for the drug which is prone to hydrolysis. The symposia focusing on the potential use of TiO2-free coatings will provide benefits to scientists from industry, academia, and regulatory agencies to help share the information/learning and navigate through the challenge being faced by the industry, if TiO2 were to be banned in pharmaceutical applications.

From Micro to Nano - PLA-PEG Block Copolymers Enable Nanoparticle Drug Products via Nanosonication for the Treatment of New Therapeutic Modalities

October 10, 2023, 12:30-2:00 pm ET

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Nanomedicine encompasses a wide variety of formulations including polymeric nanoparticles, liposomes, lipid nanoparticles, micelles, polymersomes, nano-emulsions, nanotubes, and nanofibers. These nano-sized constructs can be used to overcome drug delivery challenges such as drug solubilization, API targeting, protection of sensitive APIs from degradation and/or immune responses, and API transport across biological barriers. This webinar will focus on biodegradable polymeric nanoparticle formulations and the products and processes required for achieving sub-micron particle sizes.

Many traditional microparticle applications rely on copolymers of lactide and glycolide (LG polymers) for controlled and/or sustained release. Nanoparticles of LG polymers, while also potentially imparting some level of controlled drug release, are intended to effect targeted delivery of an API to a desired location rather than sustained systemic delivery. The amount of applied energy required to generate particles in the nanometer range can be reduced by incorporating a poly(ethyleneglycol) (PEG) block into the LG polymer chain. While traditional LG polymers can be used, the amphiphilicity imparted by PLA-PEG di-block copolymers, makes them especially suited for nanoparticle formation. By adjusting the relative molecular weight of the mPEG and poly(DL-lactide) blocks, the excipient properties and formulation properties can be carefully tuned.

While excipient quality and security of supply is paramount, the process by which nanoparticles are made is also critical to the end drug product. Current processes include high-pressure homogenization, high-shear mixing, probe sonication, and microfluidics, among others, which often result in temperature and pressure elevations that are detrimental to sensitive APIs and high-shear forces that can cause nanoparticle deformation and aggregation. The process scale limitations also inherent to these batch processes severely limit their applicability to commercial-scale and peptide/protein-based nanoparticle formulations. As a result, regulatory agencies continue to push for continuous nanoparticle processes with robust scale-up potential.

Learning Objectives: 

  • The current limitations of nanoparticle manufacturing and how to overcome them
  • The benefits of PLA-PEG block copolymers for nanoparticle formation

Speaker: Whitney Moro, Ph.D., Evonik

Optimized and Scalable LNP Composition for RNA Vaccine Delivery

October 12, 2023, 12:30-2:00 pm ET

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Lipid nanoparticle encapsulated RNA (RNA-LNP) vaccines have played a pivotal role in the global response to the COVID-19 pandemic and are now at various stages of development against a wide-range of other infectious diseases. Limited access to potent ionizable lipids, a key component in LNPs, and expertise for formulation and scale-up, present real barriers to entry in this field. To overcome these challenges, we developed and optimized a novel ionizable lipid and a corresponding LNP composition for the delivery of RNA in vaccine applications.
This webinar describes why access to potent ionizable lipids, a key component in LNPs, and expertise for formulation and scale-up are crucial for successful vaccine formulations.

Speaker: Naralie Orr, Ph.D., Precision Nanosystems

Highlighting US and European Regulatory Requirements for Gene and Cell Therapy Products

October 17, 2023, 12:30-2:00 pm ET

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Cell and gene therapy products continue to evolve, diversify and challenge the global regulatory frameworks for complex biological medicines. Nevertheless, the industry continues to mature and grow alongside both the skills within the industry and the knowledge of regulatory authorities, with the latter facing ever increasing demands upon their resources. However, in spite of this increase in experience and expertise within the community, and international efforts to increase regulatory harmonization, significant differences in both the legal requirements and scientific guidance for cell and gene therapy products inevitably remain between the major global markets.

The most obvious example of this is between the US and Europe, arguably the two most evolved regulatory frameworks for cell and gene therapy products. This webinar seeks to provide both a high-level overview of the differences between the US and European regulatory requirements for these products and a deeper dive into topics where the disparity between the two territories is greatest. This will include the way that products are classified, the regulation of clinical trials, scientific and regulatory advice procedures, the impact of the EU GMO legislation and product quality (CMC) requirements.

Patrick Ginty, Boyds
Sabine Ruehle, Boyds

In silico Modeling for Small Molecule Drug and Biologics Formulations

November 7, 2023, 12:30-2 pm ET

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In the process of developing new drugs, finding good or near-optimal solutions to challenges in drug formulation and discovery programs is essential. However, the limitations of time, expenses, and data availability often constrain the options explored in pharmaceutical applications. Here, we explore how certain computer modeling techniques can impact drug development programs by generating valuable data, often in quantities that would be impractical or impossible to obtain otherwise.

Physics and Structure-Based molecular modeling (PSB) plays utilizes the 3D structure of molecules and their interactions to generate data through a variety of techniques, including energy minimizations, conformation searches, docking, transition state prediction, and molecular dynamics simulations. Machine Learning (ML) techniques construct correlations between experimental or calculated values and more complex quantities. The impact of both PSB and ML has been steadily growing for decades within the pharmaceutical industry.

One of the main challenges in the productive application of ML lies in obtaining a sufficiently large and diverse set of training data to address the challenges at hand. However, an strong synergy emerges when PSB calculations are practical and reliable enough to systematically augment or generate large volumes of data for use in creating ML models.

This talk will present selected usage cases for each of these techniques and their synergies, showcasing their application in important areas such as:

  1. Crystal polymorph prediction
  2. API solubility
  3. pKa prediction
  4. Structure development and evolution in pharmaceutical systems
  5. Protein viscosity calculations

Speaker: John Shelley, Schrodinger, Inc.

Expanding Role of Artificial Intelligence for Drafting Regulatory Submissions

November 16, 2023, 12:30-2:00 pm ET

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Regulatory submissions, characterized by their structured data and highly templated format, are ideal candidates for drafting by generative artificial intelligence (gAI) and structured content authoring (SCA) software. By harnessing the combined power of gAI and SCA, the traditionally time-consuming and labor-intensive process of submission drafting can be dramatically expedited with concomitant improvements in quality and consistency of messaging. This webinar presentation will explore strategies for leveraging the combined power of gAI and SCA as a cost-effective tool to maximize the productivity of scarce, highly skilled and qualified regulatory writers.

Speaker: David Hauss, Ph.D., Hauss Associates, LLC

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