Contract Pharma33m
FDA Grants Orphan & Rare Pediatric Designations to Tikun’s FD Treatments
The goal is to treat familial dysautonomia patients with a single injection.
Intellia doses first subject in trial of NTLA-2002 for hereditary angioedema
The HAELO trial will assess the therapy’s safety and efficacy in 60 adult subjects with Type I or Type II HAE.