Intellia Completes Phase 3 Enrollment for HAE Gene Therapy
September 18, 2025 — Cambridge, Massachusetts Intellia has completed enrollment in the global Phase 3 HAELO trial of lonvoguran...
Orphan Drug
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Health Canada Approves Bylvay for Cholestatic Pruritus in ALGS
OTTAWA, Canada, September 9, 2025 — Health Canada has granted approval to Bylvay™ (odevixibat) for the treatment of cholestatic...
REGENXBIO Reports Positive 12-Month Data from RGX-121 CAMPSIITE® Trial in MPS II
EGENXBIO Inc. (Nasdaq: RGNX) announced positive 12-month pivotal data from its Phase I/II/III CAMPSIITE® trial of RGX-121 (clemidsogene lanparvovec),...
Zydus Therapeutics Reports Positive Topline Results from EPICS-III Trial of Saroglitazar Magnesium in Primary Biliary Cholangitis
Zydus Therapeutics, Inc., a U.S.-based innovation arm of Zydus Lifesciences Ltd., today announced positive topline data from its pivotal...
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