The introductions of Spinraza and Zolgensma in SMA offer new insights into how to address neurodegenerative diseases. But more real world evidence is needed.

Public Company Edition: Of five drug developers expected to go public, four have preclinical or early clinical candidates, while the one that could not find a market for its IPO is in Phase III. Also, Biohaven, Zymeworks and Kura raise $100m or more in follow-on offerings.

As it moves away from virology, Gilead’s signs fibrosis collaboration with Scholar Rock. Novartis seeks to acquire CMDO that manufactures Kymriah, while Intrexon regains control of CAR-T program partnered with Merck KGAA. More deals from over the holiday period… 

With its first AAV gene therapy candidate from Bamboo Therapeutics expected to report data in 2019, Pfizer’s efforts to get in the DMD competition now center on that modality.