KalVista's sebetralstat pediatric trial data heads to Madrid. What it means for the HAE on-demand market and a 2026 FDA filing. Read the analysis.
IntraBio files the first-ever FDA application for an A-T therapy. Analyse the Phase III evidence, regulatory pathway, and what approval would mean.
Sarepta's ELEVIDYS Cohort 8 targets non-ambulatory Duchenne patients with a sirolimus regimen. What the trial design reveals about the path to label restoration.
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
Curatis and Neupharma sign a CHF 83.5m Japan licensing deal for corticorelin in peritumoral brain edema. Read our analysis of what the deal means.
Quince Therapeutics ends clinical development of eDSP for A-T after Phase 3 failure. Find out what this means for its platform and future strategy.
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.
Can Aurora Therapeutics make CRISPR work at scale? Find out how its platform model for rare disease editing is testing biotech’s next big leap.
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