BridgeBio Shifts Filing Plans After Phase 3 Success in Rare Disease Drug BBP-418

BridgeBio; BBP-418; Phase 3 success; limb-girdle muscular dystrophy type 2I/R9; FDA filing; rare disease drug; NDA (New Drug Application); ribitol; full approval; biomarker; acoramidis; commercial strategy

bluebird bio Rebrands as Genetix Biotherapeutics Following Private Equity Buyout

bluebird bio; Genetix Biotherapeutics; private equity buyout; Carlyle; SK Capital Partners; gene therapy; rebranding; FDA-approved therapies; David Meek; sickle cell disease; β-thalassemia; cerebral adrenoleukodystrophy; commercial execution; manufacturing investment

Rocket Pharmaceuticals Expands Leadership with J&J and AstraZeneca Alumni to Strengthen Gene Therapy Commercialization

Rocket Pharmaceuticals, gene therapy, commercial leadership, Sarbani Chaudhuri, Johnson & Johnson, AstraZeneca, commercial launch, gene therapy market, rare diseases, biotechnology, Kresladi, RP-L102.

Lexicon Pharmaceuticals Shifts Focus to Clinical Development Following FDA Setbacks

Lexicon Pharmaceuticals, FDA setbacks, Clinical development focus, Commercial operations elimination, Sotagliflozin, Type 1 diabetes, Chronic kidney disease, Restructuring

Vertex’s CASGEVY Marks Historic Milestone with First Commercial Patient Infusions, Paving the Way for 2025 Launches

CASGEVY, Vertex Pharmaceuticals, gene therapy, sickle cell disease, beta thalassemia, CRISPR/Cas9, FDA approval, commercial patient infusions, 2025 launches.