Webinar Recap: CRISPR Genome Editing Solutions from Discovery to Clinic 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.   ..read more

Seeking guidance for a big investment 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.  ..read more

Expanding the Public Health Impact of Gene Editing 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 ..read more

Considering the processes of mRNA manufacturing 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.  In the case of Contract Development and Manufacturing Organizations (CDMOs), it is like a car, very practical for getting you from one point to another. They are ..read more

Making use of ultra-low endotoxin Cas9-RNPs from Aldevron 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-RNPs* for IGI to enable a novel in vivo CRISPR delivery system, targeted directly into the brain for editing.  ..read more

Working toward advanced analytical techniques 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 ..read more

AAV ITRs versus Transposon ITRs 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.  ..read more

Celebrating the work ahead On my flight to Boston for the mRNA-Based Therapeutics Summit, I followed my usual routine as an uneasy flier; put headphones on and tune out until landing. This time, however, the music continued as I found certain lyrics of one particular song repeating in my mind throughout the two-day gathering of industry leaders ..read more

Streamlining the mRNA manufacturing process from Sequence-to-Vial 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 ..read more

Validation of mRNA concentration determination using variable pathlength spectroscopy Analytical testing is a critical factor in the development of mRNA-based therapeutics, while at the same time presenting significant challenges. Pre-clinical and clinical testing are only part of what needs to be included in the development process, with the need to meet regulatory requirements while competing in the marketplace being additional considerations.   ..read more