Rein Therapeutics Receives FDA Fast Track Designation for LTI-03 in Idiopathic Pulmonary Fibrosis
Phase 2 RENEW trial underway; interim data expected in H2 2026.
Phase 2 RENEW trial underway; interim data expected in H2 2026.
AX-0810 Phase 1 shows dose-dependent bile acid increases and no serious adverse events, delivering the first human proof of concept for ProQR’s Axiomer RNA editing.
Cantor Fitzgerald initiates Overweight coverage on Milestone, citing Cardamyst’s strong PSVT launch potential and $250-300M peak sales outlook.
Ultragenyx’s GENGLYCOS receives accelerated FDA approval as the first gene therapy targeting the root cause of GSDIa in patients aged 8 and older.
BMO Capital starts coverage on ALNY, ASND, BMRN, CATX & more with Outperform ratings and price targets. See full analyst insights and targets.
Sellas GPS data is imminent, Maxim keeps Buy/$30 PT. BNTX also in focus.