The partnership worth up to $1.75bn removes the significant risk of going it alone in a competitive market as Regenxbio awaits late-stage data for its gene therapy.

Public Company Edition: This year is just six initial public offerings away from matching 2020’s record-breaking total of 86 biopharma IPOs in the US. Also, Idorsia raised $662.5m through the sale of convertible bonds and Cytokinetics closed a $316.3m follow-on offering. 

Denali Therapeutics has reported promising early clinical data in Hunter syndrome for its fusion protein, DNL310, an investigational agent that crosses the blood-brain barrier and is expected to address disease-associated CNS effects; however, some biomarker data disappointed, and there are potential competitors also under development.

Adverum said it would cease developing ADVM-022 for DME and focus on wet AMD, but the adverse events that prompted the shift could invite further scrutiny in an already competitive market.