Argo Biopharmaceutical Co., Ltd. (Argo Biopharma), a Shanghai-based clinical-stage RNA interference therapeutics company, has received Fast Track Designation from the U.S. Food and Drug Administration for BW-20805, its investigational small interfering RNA therapy targeting prekallikrein for the prevention of hereditary angioedema attacks. The designation comes as the company runs a global Phase II open-label study […]
Can SonoThera’s ultrasound platform reshape gene therapy delivery? Find out how its $125M Series B targets DMD, ADPKD, and XLAS with nonviral precision.
KalVista’s oral HAE drug EKTERLY hit $49M in 2025 sales. Discover what strong refill demand and global expansion mean for rare disease treatment models.
GenSight Biologics S.A.’s early access rollout of GS010/LUMEVOQ in France and Israel has drawn new attention to how regulators are quietly repurposing compassionate use frameworks into strategic regulatory sandboxes for advanced therapies. As rare disease gene therapies edge closer to commercial viability, a patchwork of early access programs across key jurisdictions is beginning to influence […]
