| |   | | | Messenger molecule with ‘copy machines’ The mRNA molecules used to ferry the Covid vaccine throughout the body have a short shelf life. Although scientists have worked to improve their stability, they've so far come up short. And that’s not necessarily a bad thing — for gene therapies, it wouldn’t be great if DNA-cutting enzymes ran amok indefinitely after they’ve accomplished their intended edit. But there are other situations in which patients might need to produce higher levels of a therapeutic protein — making the current mRNA tech a bad fit. That’s why Replicate Bioscience is developing messenger RNA that makes more of itself once it’s adopted by cells. This self-replicating RNA can work because it codes for proteins normally found in viruses which allow them to copy their RNA genomes once they’re inside cells. In essence, these mRNA molecules have their own in-built copy machines. Read more. | A setback for Editas Medicine Editas Medicine will be halting its first clinical program after data showed only three out of 14 patients receiving its CRISPR-based therapy showed “clinically meaningful” improvements. The treatment, called EDIT-101, is geared toward a rare genetic eye disease called CEP290-mediated LCA10. The company says that the reason three patients responded was because two had two — as opposed to one — copies of the mutation that causes the disease. But the company said only 300 people have that same genetic profile, which was too small a market. Editas has faced several setbacks in recent years, lagging behind rivals. Its only remaining clinical program is EDIT-301, an experimental treatment for sickle cell disease. But it’s only in early clinical development, while rival Vertex is preparing to file for FDA approval for its own sickle cell gene therapy. Read more. | STAT E-Book: The digital technologies transforming clinical trials Technology is spurring rapid advances in how we diagnose and treat disease. It’s also transforming how scientists run clinical trials. But that promise is only as good as the quality of the data researchers collect, and the technical infrastructure they can use to make sense of it. Our latest e-book reflects the growing potential of technology in clinical research, as well as the hurdles that stand in the way of its broader use. Download now. | Biosimilar uptake needs a shot in the arm Biosimilars are still underused in the U.S., despite their potential to lower drug costs. Here’s why: There are hurdles to market access, and there's a stigma that biosimilars are inferior to their branded counterparts, opines Thomas Newcomer, an exec at biosimilar maker Samsung Bioepis. Meanwhile, biosimilars are faring much better in Europe. This is because of marked differences between the E.U. and U.S. patent landscapes. The U.S. also has a far more fragmented health care system than most European countries. But patients in the U.S. in particular seem not to understand there are no clinically meaningful differences between reference biologics and their biosimlar equivalents. Breaking down some of these barriers, Newcomer says, can “provide a financial pressure valve that allows the health care industry to invest in future innovations.” Read more. | Roche’s failure, Eisai’s success, and a live AMA How do Alzheimer’s drugs even work? Can biotech people bake? And do we even like one another? We cover all that and more this week on “The Readout LOUD.”
Recorded live from the 2022 STAT Summit, we discuss the failure of an Alzheimer’s disease treatment from Roche, the unexpected success of a competing one for Eisai, and some unpredictable questions from our audience.
Listen here. | More reads - Convicted Theranos founder Elizabeth Holmes to be sentenced Friday, Wall Street Journal
- FDA wants confirmatory trials to be underway before granting accelerated cancer nods, Pazdur says, FierceBiotech
- Big Pharma may have to reveal government deals in WHO's draft pandemic rules, Reuters
| Thanks for reading! Until next week,  | | | |
No comments