Nomlabofusp program granted Breakthrough Therapy Designation for the treatment of adults and children with FA based on FDA’s review of available ...

STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) ...

Recurrent respiratory papillomatosis (RRP) is a rare HPV-related disease of the respiratory tract with significant unmet need INO-3107 previously received Orphan Drug and Breakthrough Therapy ...

The Food and Drug Administration (FDA) development and approval process for drugs and medical devices involves multiple phases that ensure the safety and effectiveness of products before and after ...

RICHMOND, Calif., Nov. 21, 2025 (GLOBE NEWSWIRE) -- Sangamo Therapeutics, Inc. (SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has accepted Sangamo ...

PDUFA action date set for August 23, 2026 If approved, DTX401 will be the first treatment to address the underlying cause of GDSIa NOVATO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Ultragenyx ...

Savara (NASDAQ:SVRA) executives provided an update on the company’s regulatory timeline, manufacturing changes, clinical evidence, and commercial preparations for MOLBREEVI during a presentation at ...