Vironexis’ AAV tech to express protein therapeutics; plus updates from Rarity, Mendus and more

A group led by Timothy Cripe at Nationwide Children’s Hospital designed a recombinant adeno-associated virus (AAV) vector to express bispecific T cell engager Blincyto blinatumomab from  Amgen Inc. (NASDAQ:AMGN) as an off-the-shelf, single-dose option to sustain systemic serum levels of protein immunotherapeutics for at least one year.

The team also developed an inducible version for conditions that require either short-term or recurring, intermittent expression of a therapeutic. The construct contains two introns flanking a defective exon in the transgene coding sequence that represses expression of the transgene until administration of a morpholino complementary to the 3′ exon splice site...