Gene-modified cell therapies have been an area of much focus for drug developers over the last decade in the cell and gene therapy arena. Innovations around CRISPR technology have made it faster and more straightforward than ever before to manufacture patient doses of gene-modified cells.
Messenger RNA (mRNA) based drugs and vaccines make up a new class of medicine showing tremendous promise. Compared to established drug and vaccine technologies, mRNA has the potential to reach patients faster by being easier to develop and produce.
CRISPR-Cas shows promise in leveraging nonclinical and manufacturing information from one disease indication to another, potentially without repeating redundant, costly, and time-consuming nonclinical studies. That’s the driving message in a webinar that featured a pioneer in human genome editing, Dr. Fyodor Urnov, Scientific Director at the Innovative Genomics Institute (IGI).
Biotech Week Boston is always an exciting time, with so many events happening at once. Vendors and participants buzz around to decide what presentations and meetings to attend, and which vendors they want to see. During the event, Aldevron had two highlighted speaking slots that attracted a good amount of attention.
Similar to a comedian developing a stand-up routine around the latest trends, a Chief Scientific Officer (CSO) drives current methods and technologies for biological therapeutics. Both have insightful viewpoints of their trade, with the CSO closely working in the world of both academic and industrial research for development of drug products from conception to commercialization.
The latest research article from the Innovative Genomics Institute (IGI) at the University of California, Berkeley, highlights the collaboration between Aldevron with the laboratories of Nobel Laureate Dr. Jennifer Doudna and Dr. David Savage. Published in Molecular Therapy-Nucleic Acids, “Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs,” shows how Aldevron generated ultra-low endotoxin Cas9 for IGI to enable a novel in vivo CRISPR delivery system, targeted directly into the brain for editing.
Guten tag from Berlin! As an analytically minded-person, it’s fascinating to connect with colleagues from around the world to discuss cutting-edge techniques and strategies for ensuring the quality, efficacy, and safety of mRNA-based therapeutics. That fascination led me to the mRNA Analytical Development Summit, held in Germany's capital city.
Inverted Terminal Repeats (ITRs) are DNA sequences flanking the transgene of interest to signal incorporation into adeno-associated virus (AAV) or genome insertion by transposases. Despite sharing the ITR element name, viral vector and transposon ITRs differ in sequence and important properties that need to be considered in handling these vectors.
The manufacture and release of mRNA therapeutics and vaccines is a complex and challenging process requiring a high degree of expertise, capabilities, and experience. The development and manufacturing of final drug product involves several stages that include production of cell banks, linear DNA, and mRNA drug substance, with lipid nanoparticle (LNP) formulation/encapsulation and analytical testing at all steps, through aseptic fill/finish.
