Blog – Page 3043

One of the oldest known molecules, the ribosome, creates proteins based on a copy of the genetic code found in the genome, known as mRNA. Scientists have believed that the ribosome performed the same type of work with all mRNA, like a standardized assembly line that it did not regulate on its own. However, researchers from the University of Copenhagen have discovered this is not the case.

Their findings are published in the journal Developmental Cell in an article entitled “Ribosomal RNA 2′-O-methylation dynamics impact cell fate decisions.”

“It has long been known that there are different types of ribosomes. But it has been assumed that no matter what mRNA you give the ribosome, it will produce a protein. But our results suggest that different types of ribosomes produce specific proteins,” says Anders H. Lund, professor at the Biotech Research and Innovation Center at the University of Copenhagen.

Recombinant adeno-associated virus (rAAV) vectors are a cornerstone of genetic medicine. These hollow virus particles are used to deliver commercial gene therapies like Glybera and Luxturna, as well as many other candidates still in development.

Despite this, the biopharmaceutical industry has yet to find the most effective way of making rAAV vectors at scale. McKinsey analysts recently described vector production as one of the major challenges faced by gene therapy developers. And the situation is only going to get worse as the gene therapy market evolves and begins developing products for larger patient populations.

“With a shift beyond ultra-rare indications, viral-vector manufacturing requires rapid expansion to be able to address various diseases in the commercial space,” the analysts wrote, adding, “The broader application of viral vector-based gene therapies (for example, to more common diseases) requires higher yields and lower cost of goods (COGs).”