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. 

AGTC hopes to gain the first approval in X-linked retinitis pigmentosa for its gene therapy AGTC-501, now in a Phase II/III study. It also is advancing development in achromatopsia, another unmet medical need.

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.