Though Marks emphasized that this concept/designation at the regulatory level is still somewhat of a “we’ll-know-it-when-we-see-it” phenomenon, his remarks this summer — as well as a few additional discussions that have occurred throughout 2023 — add a bit more clarity to our understanding of what a gene therapy platform can be.
Ultra-Sensitive Assay For Anti-Sense Oligonucleotide Quantification
Learn about a new level in sensitivity for challenging analytes in matrix that opens doors for accurate bioanalysis and metabolism studies with specificity not achievable with orthogonal methods.
Pre-Banking And Avoiding Manufacturing Challenges For GMP Plasmids Containing Unstable Sequence Regions
Ensuring consistent replication and proper retention of plasmid DNA sequence is vital to the economics and efficiency in generating starting materials used for production of AAV delivered gene therapy and mRNA vaccines and therapeutics.
Boost LNP Drug Potency With Optimized Delivery Formulations
Lipid nanoparticles (LNPs) function as a drug delivery system for nucleic acids. Learn about the biomedical applications of LNPs as well as the anatomy, formulation optimization, and safety of LNPs.
Analytics And Bioassays Can Safely Fast-Track mRNA-LNP Drug Development
One main mRNA technology challenge is the formulation of delivery systems like lipid nanoparticles (LNPs). Learn about evading impurities in lipids, the future of mRNA-based genomic medicines, and more.
RNA: The Next Era In Therapeutic Development
Review the unique set of challenges for RNA production that must be considered and solved to reach their full therapeutic potential.
RNA Biotherapeutics — A New Approach To An Old Problem
Check out a brief history of RNA-based therapy and how Droplet Digital™ PCR supports this field of work.
Common Approaches To Bioanalysis Of Oligonucleotides
Weigh an assortment of advantages and disadvantages to determine which strategy is best for a clinical study.