Happy Eve of Not #JPMorgan: here are my in-no-particular-order Top 10 predictions for the year in cell and gene therapy, not so much as a Nostradamus impression, but hopefully more a provocation of thought…
#1 The investment and acquisition arms race in the CTx and GTx tools & technology space will continue, with valuations that baffle us all, especially for CMOs
#2 mRNA-nanoparticle technology will become regarded as a bona fide GTx, expand out of its world-changing year in vaccinology, and comprise the first for-real competitor to viral vector technology
#3 Regulators will continue to drop the hammer on CMC, especially, but not limited to, potency assays: sponsors will howl they are suddenly ‘tightening up,’ but cooler heads will remind the CGTx field it’s simply maturing to a point where regulators have always been strict
#4 What’s going on with toxicity and high-dose AAV will be clarified—and it won’t be good news for high-dose AAV. Capsid engineering could save the day, but only if it actually shows effectiveness of reduced doses
#5 The myth of mesenchymal stem cells being all-seeing, all-knowing, anti-inflammatory, ‘smart cells,’ ‘cells that know what disease needs to be treated and do what needs to be done,’ will finally die: everyone, we wish they did, but they don’t work
#6 Allogeneic CAR-T, NK etc. therapies will either show a definitive CMC advantage (= consistently high doses/donor) or risk being considered insufficiently differentiated from their autologous competitors
#7 For the first time in 20 years, scale-up and consistency of Adenovirus (yes, not the Associated Virus) will become really important—and not just for viral vaccines
#8 CGTx clinical assets will become commodities, in a good way, enabling companies with rich and diverse pipelines to monetize and streamline
#9 Scott Gottlieb, M.D. will return as FDA Commissioner, to a ticket tape parade and rows of CoVID-19-vaccinated staffers prostrating at his feet and the sound of a chorus of angels (just kidding about the angelic choir – umm, kind of – but well done for reading this far)
And #10 (this one’s for 2022): hotel room rates in the second week of January in San Francisco will drop back into the triple digit $. I just hope there’s still a #JPMorgan conference there for us to avail of them…