MANUFACTURING ARTICLES

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.
MANUFACTURING RESOURCES
-
Both physical and chemical nonviral gene delivery systems for ex vivo genetic modification offer advantages over viral vectors, including smaller scale production and the low risk of immunogenicity.
-
Explore challenges to the successful development of RNA-based drugs and vaccines as well as the advantages of utilizing lipid nanoparticle technology as a delivery platform for saRNA.
-
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.
-
Learn how a strong anion exchange resin can be used for the efficient purification of single-stranded DNA oligonucleotides used in clinical trials.
-
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.
-
In this scientific brief we evaluate the performance of the the Pall Nucleic acid binding (NAB) Nanosep centrifugal device for nucleic acid isolation.
-
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.