Predictions over who would win the Nobel Prize for CRISPR have raged for several years. While some speculated that the award might come from the Physiology or Medicine committee, CRISPR-based therapeutics have only just entered the clinic. It will be many years before we see significant medical impact from the technology. That put the spotlight on Chemistry. There was little doubt that, should a Nobel Prize for CRISPR be awarded in Chemistry, Doudna and Charpentier would claim two of the possible three slots.
Every new biotech firm hopes to succeed like Genentech, the original biotech startup. Today, several new firms are focused on gene therapy challenges such as vector selection and manufacturing.
Besides addressing lingering safety concerns, gene therapy is exploring applications beyond the correction of single-gene mutations. Possibilities include polygenic and gene-independent approaches.
Viral vectors offer efficient delivery. (But they’re cramped and potentially pathogenic.) Nonviral vectors privilege safety. (But they’re relatively inefficient.) Fortunately, both are improving.
If you like GMO patchwork, stick with transgenic crop development. But if you want fine tailoring, embrace gene editing. It enhances a plant’s own qualities and has a natural, sustainable look.
In research, the demand for DNA strands often outpaces supply. Researchers needing a few genes have a choice of several providers. But what if researchers need 10,000 genes? “We’re probably the only game in town,” suggests Emily Leproust, PhD, co-founder and CEO of Twist Bioscience. Twist has developed a synthetic DNA manufacturing process that emulates the miniaturization seen in the semiconductor industry.
Oxgene maintains that TESSA—a Tet-enabled self-silencing adenovirus—can change theway that AAV vectors are manufactured, reducing costs, improving quality, and promoting safety.
Refining the delivery method so that the desired gene reaches the right part of the brain in the right amounts can be especially challenging in larger animals that are the key to obtaining data that translates to the clinic. Choosing to work with a seasoned research partner like Charles River Laboratories provides access to decades of experience at every step of the process.
In response to calls for more inclusive participation in the governance of genome editing technologies and their applications, a Policy Forum has proposed the creation of a global citizens’ assembly. Composed of a minimum of 100 lay citizens from around the world and informed by experts, the assembly would explore and deliberate the implications of genome editing technologies and regulatory principles.
Catalent’s third gene therapy facility at Harmans, MD, will use a sophisticated infrastructure that is able to gather, manage, and analyze data across all operations.