Industry News update covering July 2021

Peter Timmins

摘要

Quite a number of news items this month included cell/gene therapy and RNA/oligonucleotide therapeutics stories. These included a new gene therapy becoming available from bluebird bio, as well as the return to the market of a product from that company after it had been voluntarily suspended to allow for a suspected safety issue to be resolved. Others had less positive news, like Adverum which unfortunately had to terminate trials of its ophthalmic disease gene therapy due to serious safety concerns. RNA-based therapeutics included Dicerna’s new antisense for rare liver disease and Arrowhead’s gene silencing agent for kidney cancer showed favorable Phase I trial outcomes. However, Arrowhead’s cystic fibrosis RNA interference drug had preclinical safety concerns that paused an early phase trial. Bicycle Therapeutics and Ionis Pharmaceuticals agreed a collaboration to develop tissue-specific oligonucleotide therapeutics using cyclic peptide targeting while Moderna moved its mRNA vaccine technology on from COVID-19 to explore a multivalent influenza vaccine. Still in large molecule space, some interesting biologics-enabling technologies were announced with Lilly accessing glucose-sensing insulin approaches, VitriVax gaining funding to advance its thermostable vaccine technology and Prime Medicine taking a novel gene editing approach forward with its combined fusion protein editor/RNA guide and replacer template complex delivered via a lentivirus vector technology. Small molecules also were still bringing therapeutic advances, either well-known molecules through drug delivery technology into new indications (e.g., budesonide-targeted modified release capsule from Stada/Calliditas) or with novel molecules against novel targets (e.g., Ribon’s PARP7 inhibitor). Drug delivery technology companies were the target of acquisitions, perhaps most significantly with cigarette maker Philip Morris stepping into the delivery technology space in buying Fertin Pharma and bidding to acquire Vectura.

Industry News update covering July 2021

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