Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium and Oxford BioMedica aim to get their CFTR gene therapy into the clinic soon, confident it could offer a long-lasting option for patients with cystic fibrosis.

Some remain unconvinced on durability, but CRISPR Therapeutics is looking at a second ‘consolidation’ dose and other measures to boost CTX110’s efficacy in a pivotal trial.

ARM counts $14.1bn in fundraising in the first half of 2021, up 35% from mid-year 2020. Also, Chinese mRNA players Abogen raises a $700m series C venture capital round, Pfizer prices a $1bn sustainability bond and while IPOs slow in the US, China and South Korea see new first-time offerings.

Intellia’s announcement of the first in vitro gene editing proof of principle sent shockwaves through the life science investment space. But are there still many known unknowns.