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.

Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.

Investigational products from Ultragenyx and MinervaX secured a place on the European Medicines Agency’s priority medicines scheme this month.

Ultragenyx is buying its Angelman syndrome partner GeneTx for $75m on the heels of an interim Phase I/II readout that shows signs of clinical activity and appears to resolve earlier safety worries.