NEW YORK, NY — Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to NGN-401, an investigational gene therapy in late-stage clinical...

SAN DIEGO, Calif. — ADARx Pharmaceuticals, Inc. (ADARx), a late-stage clinical biotechnology company developing next-generation RNA therapeutics, today announced Phase 1/2 clinical data for onvuzosiran (ADX-324), an investigational small interfering RNA (siRNA) therapeutic candidate being developed for the treatment of hereditary angioedema (HAE). The Company also highlighted the clinical trial design...

AMSTERDAM, the Netherlands — Norgine B.V., a leading European specialty pharmaceutical company, today welcomes the positive opinion issued by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommending the granting of a marketing authorisation under exceptional circumstances[*] for XOLREMDI® (mavorixafor) in patients aged 12 years and older for...