The morning session will focus on comparability for novel protein-based modalities. The afternoon session will focus on comparability for mRNA vaccines and microbiome products.
Innovative 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.
Innovative 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.
The afternoon session will focus on comparability for mRNA vaccines and microbiome products.
Mass 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.
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.
Cell 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.