Partners pioneer approach in rare disease in what will be pivotal year for CRISPR and gene-editing technologies.

Public Company Edition: Phathom went public in the US within proposed pricing terms, but Cabaletta’s IPO went to market below a proposed range, while TFF launched in line with earlier plans. Also, Principia’s FOPO raises $210m and Trillium restructures.

How did a youthful vendor of IT services, faced with a personal exposure to crippling illness, attract some of the best minds in biotech to launch a company with a then-untested RNA technology that today is a contender for leadership in the rarified space of inherited retinal disease? The answer may lie in the ambitiously definitive name that 36-year-old founder Daniel de Boer gave to his enterprise with a mission: ProQR – an active synonym for finding cures.

Public Company Edition: COO Dean Hum talked to Scrip about Genfit's IPO strategy – which wasn't just about raising money – as it anticipates Phase III elafibranor data later this year. Also, Precision BioSciences launches its IPO, while Myokardia, Dermira and Tricida lead new public company financings.