CMC Strategy Forum North America/WCBP 2023
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DateJanuary 23, 2023The morning session will focus on comparability for novel protein-based modalities. The afternoon session will focus on comparability for mRNA vaccines and microbiome products.
DateJanuary 23, 2023Innovative approaches in the formulation of biologics will be shared in this session. Topics covered will include exploring the strategies in the development of formulations to support co-formulation of drug products and different routes of administration. The use of nanoparticles in the development and delivery of mRNA therapeutics as well as the use of application of novel freezing and drying technique will be explored. The session will also include an interactive panel discussion following the presentations.
DateJanuary 23, 2023Innovative approaches in the delivery of biologics will be shared in this session. Topics covered will include the use of exosomes in the delivery of oligonucleotides as well as the combination of device technology with oral delivery of biologics. Insights into the use of a Proteo-Lipid Vehicle platform for the delivery of therapeutics will also be shared. First genetic medicines platform to enable highly targeted delivery to organs, tissues and cells beyond the liver will be discussed. The session will also include an interactive panel discussion following the presentations.
DateJanuary 23, 2023The afternoon session will focus on comparability for mRNA vaccines and microbiome products.
DateJanuary 23, 2023Mass photometry is a single-particle analytical technology that measures the masses of biomolecules in their native states, in solution. The TwoMP and SamuxMP mass photometers are easy-to-use instruments that can measure masses of biomolecules between 30 kDa and 5 MDa, or AAV empty/partial/full ratios, requiring minimal sample and minimal sample prep for analysis. In this talk, we demonstrate the utility of the TwoMP as a tool to measure critical quality attributes of your samples, or to quickly, easily monitor antibody-antigen interactions, quantify small-molecule induced changes to complex formation, assess sample purity and more. We wil also discuss capabilities of the SamuxMP in characterizing AAV samples. We will show that it can differentiate between empty, partially filled and full AAV capsids, and that is serotype agnostic. The SamuxMP measurements are in line with gold-standard techniques such as TEM and AUC.
Speaker
Refeyn Inc
DateJanuary 24, 2023
DateJanuary 24, 2023
Laura Sepp-Lorenzino, Ph.D. joined Intellia Therapeutics in 2019 as Chief Scientific Officer and is responsible for Research and Early Development. Intellia is harnessing CRISPR-based technologies to revolutionize the future of medicine. Laura previously held leadership positions at Vertex, Alnylam and Merck. She serves in the Board of Directors of Taysha Gene Therapies, the Alliance for Regenerative Medicine and the Oligonucleotide Therapeutics Society, and on the Scientific Advisory Boards for Thermo Fisher Scientific, the U.K. Nucleic Acid Therapies and Arsenal Capital Partners. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina, and both her M.S. and Ph.D. in Biochemistry from New York University.Speaker
DateJanuary 24, 2023Cell and Gene Therapy Products (CGTP) have been in development for the last 30 years and the recent the advances in basic science, manufacturing technologies and supported by regulatory framework have paved the way commercialization of CGTPs. These advanced therapies have enabled the successful targeting of monogenic diseases using in vivo corrective technology (e.g., adeno-associated vector, AAV) and treat cancer using ex vivo therapies based on genetic engineering of patient cells prior to the re- infusion (e.g., CART products for blood cancers).
This plenary will provide insight into the development these novel cutting edge CGTP therapies having the potential to further advance the targeted treatment of patients’ unmet medical needs. Specifically, the session will focus on innovative therapies based on genome editing, progress in regenerative medicine, and new viral vector design. In addition, the session will address the current regulatory expectation for these novel CGTP therapies.
DateJanuary 24, 2023While the implementation of ICH Q12 tools and enablers across the US, EU, Japan, Canada, Switzerland and other ICH member countries is under way, it is recognized that the global regulatory authorities are at different stages of incorporation of ICH Q12 principles and tools, which in itself can pose challenges. This session will be focused on real life examples pertaining to ECs, PACMPs, PLCM documents and the PQS from global ICH Q12 related pilot programs and submissions to health authorities to help understand the benefits and challenges associated with this enhanced framework. The understanding gained would facilitate effective, predictive management and operational flexibility of post-approval lifecycle changes thus enabling timely access of high quality, safe, and effective medicines to patients while reducing unnecessary cost, time, and burden on industry and regulators.Speakers
DateJanuary 24, 2023The COVID-19 pandemic demonstrated that international collaboration among global regulatory authorities and with industry is essential, achievable and enables the best use of available resources and expertise. Regulators and industry collaborated to expedite product development and decision-making processes to enable treatment availability at an unprecedented pace, scale, and geographical reach.
What are our lessons learned? How can we leverage the experiences gained during the pandemic to streamline development, harmonize systems, and drive faster approvals to deliver quality medicines to more patients faster not only in preparation for the next pandemic but also as a new way of working?
This regulatory panel will bring together regulators from several global regions for an interactive discussion about evolving practices and initiatives to support agency-agency, agency-industry and industry-industry collaborations, including:
- Discussions on the role of regional and international harmonization and regulatory convergence initiatives, including a wider implementation of international guidelines e.g., ICH, WHO, and their updates to address advances in pharmaceutical science, technology, analytics, data management, and regulatory concepts
- Share experiences from collaborative reviews, e.g., ICMRA pilots, project Orbis/Access Consortium and other collaborative pathways, discuss traditional and alternative approaches to inspectional assessment, and outline next steps to foster the use of reliance pathways
- Explore how we can accelerate regulatory harmonization not only for initial marketing authorizations but also for post-approval changes to achieve the blue-sky vision “One dossier, one submission, one inspection, one approval”, e.g., ICH M4Q revision, CMC dossier content alignment, ICH Q12 benefits.
Session Panelists:
Samvel Azatyan, World Health Organization
Christopher Downey, CDER, FDA
Veronica Jekerle, European Medicines Agency
Ingrid Markovic, CBER, FDA
Elkiane Rama, ANVISA, Brazil
Dean Smith, Health Canada
Eric Karikari-Boateng, Food and Drugs Authority, Ghana
Yasuhiro Kishioka, PMDA-Pharmaceuticals and Medical Devices Agency, Japan- DateJanuary 24, 2023
The COVID-19 pandemic demonstrated that international collaboration among global regulatory authorities and with industry is essential, achievable and enables the best use of available resources and expertise. Regulators and industry collaborated to expedite streamlined product development and decision-making processes to enable treatment availability at an unprecedented pace, scale, and geographical reach.
What are our Lessons learned? How can we leverage the experiences gained during the pandemic to streamline development, harmonize systems, and drive faster approvals to deliver quality medicines to more patients faster not only in preparation for the next pandemic but also as a new way of working?
This regulatory panel will bring together regulators from several global regions for an interactive discussion about evolving practices and initiatives to support agency-agency, agency-industry and industry-industry collaborations, including:
- Discussions on the role of regional and international harmonization and regulatory convergence initiatives, including a wider implementation of international guidelines, e.g., ICH, WHO, and their updates to address advances in pharmaceutical science, technology, analytics, data management, and regulatory concepts;
- Share experiences from collaborative reviews, e.g., ICMRA pilots, projects ORBIS/ACCESS and other collaborative pathways, discuss traditional and alternative approaches to inspectional assessment, and outline next steps to foster the use of reliance pathways; and
- Explore how we can accelerate regulatory harmonization not only for initial marketing authorizations but also for post-approval changes to achieve the blue-sky vision "One dossier, one submission, one inspection, one approval", e.g., ICH M4Q revision, CMC dossier content alignment, ICH Q12 benefits.
