Pharming Group drug Joenja was awarded FDA approval for treating a primary immunodeficiency called activated phosphoinositide 3-kinase delta syndrome, or APDS. It’s the first treatment for the rare disorder.
RNA therapies developer ProQR Therapeutics is eying a future without ophthalmology. After receiving European Medicines Agency feedback for its lead program for a rare eye disorder, the biotech has decided to find a strategic partner for all of its eye drug assets and apply its RNA technology to other therapeutic areas.
By reducing administrative burden and redesigning workflows around human needs, it creates space for what matters most: connection between clinicians and patients.
The lead RNA therapy from ProQR Therapeutics has failed the main and secondary goals of a pivotal study testing it as a treatment for a rare, inherited vision disorder. Despite the disappointing preliminary data, the biotech said it remains confident in the technology that produced the experimental therapy, sepofarsen, and the Phase 2/3 study will continue.