Why BridgeBio Pharma, Inc.’s BBP-418 priority review could redefine the future of LGMD treatment
BridgeBio Pharma, Inc.’s BBP-418 wins FDA Priority Review for LGMD2I/R9. Discover what this could mean for rare muscular dystrophy treatment.
Pharma & Biotech
Pharma & Biotech
How Tenaya Therapeutics, Inc. is positioning TN-401 against the limitations of current ARVC care
Could TN-401 change ARVC treatment strategy? Explore what Tenaya Therapeutics, Inc.’s RIDGE-1 data means for cardiac gene therapy.
Pharma & Biotech
Catalent’s Elpida Therapeutics deal shows why AAV manufacturing is still a strategic choke point
Ultra-rare gene therapies need more than science. Elpida’s Catalent deal tests whether AAV manufacturing can keep patients within reach.
Pharma & Biotech
What Soligenix’s Q1 update reveals about biotech risk after a failed confirmatory trial
HyBryte’s setback leaves Soligenix with cash, orphan assets, and a harder question. Can SGX945 reset the rare disease story?
Pharma & Biotech
Why Blackstone’s $250m Anagram deal could reshape pancreatic enzyme therapy
PERT is old, essential and burdensome. Blackstone’s $250m Anagram bet asks whether ANG003 can reset enzyme therapy.
Pharma & Biotech
Travere Therapeutics’ $475 million raise puts spotlight on rare disease execution risk
Rare disease pipelines need time and cash. Travere’s $475 million debt deal shows how biotech balance sheets are being rebuilt.
Pharma & Biotech
Rezolute’s ersodetug data deepens the congenital hyperinsulinism regulatory debate
Rezolute, Inc. has presented expanded Phase 3 sunRIZE data for ersodetug in congenital hyperinsulinism at the Pediatric Endocrine Society Annual Meeting, adding continuous glucose monitoring outcomes and open-label extension observations to a program that previously missed its primary endpoint. The update places the late-stage rare disease biotech firm in a complex regulatory position, as the