Data showed efficacy limited to IVS26-mutant Leber congenital amaurosis 10, which affects only 300 patients in the US. The company is pausing enrollment as it seeks a partner.

Share prices for many gene-editing companies declined in 2021, but the field is burgeoning with novel approaches due to be highlighted at the J.P. Morgan meeting, and one player, Intellia, could move closer to a breakthrough in 2022.

The French biotech has managed to bag a blue-chip partner to advance its ambitions to be the leading company in the ocular genomic medicine space, getting access to Intellia's CRISPR/Cas9 technology.

Patient selection is held out as the Achilles heel of clinical trial success. But a surprise and fortunate finding in Pfizer’s gene therapy trial has already resulted in tightened recruitment criteria, thanks to genetic analysis.