Partnered with AbbVie on a wet AMD gene therapy, Regenxbio is also advancing wholly owned candidates for Duchenne muscular dystrophy and MPS I and MPS II.

Also, Cytovia will go public via SPAC merger, Nkarta capitalized on its positive early NK cell therapy data with a $230m offering, and venture capital mega-rounds include $220m for Reify, $110m for Satellite Bio and $100m for OMass Therapeutics. 

An increasing number of sophisticated tools are on offer to address new therapeutic challenges and remaining unmet needs.

Gene therapy remains attractive when data show new technologies can advance the field, but gene regulation, editing and RNA therapies are grabbing investors’ attention, panelists said at ARM’s Cell and Gene Meeting on the Mesa.