Rare Disease & Gene Therapy — July 27, 2026
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AstraZeneca’s Ultomiris missed its Phase 3 primary endpoint in transplant-associated thrombotic microangiopathy, yet resilient rare disease and oncology sales kept its $80 billion 2030 revenue target intact — the same day Sarepta handed the CEO reins to Michael Severino amid an ELEVIDYS reset.
Today’s top developments:
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What to Watch
- AstraZeneca’s $80B 2030 target — With Phase 3 misses stacking up for Ultomiris in HSCT-TMA, Imfinzi in adjuvant hepatocellular carcinoma, and Wainua in ATTR-CM, watch whether Alexion’s continued pediatric Ultomiris filings and 36% Strensiq growth sustain the rare disease momentum management is banking on.
- Arrowhead’s YOSEMITE readout — Phase 3 enrollment in homozygous familial hypercholesterolemia is complete with study completion projected for mid-2027; the zodasiran data will anchor the RNAi segment’s NDA timeline and test Regeneron’s evinacumab franchise.
- Sarepta’s ELEVIDYS reset — Incoming CEO Michael Severino inherits a roughly $3.2 million gene therapy pulled and then FDA-restricted after two patient deaths; the August 5 earnings call should signal whether he renegotiates payer coverage or pushes label expansion into non-ambulatory Duchenne patients.
- Oak Hill Bio’s Angelman disclosure — As a newly SPAC-listed public company competing with Ultragenyx and Ionis, Oak Hill will need to reveal its specific modality and preclinical data in upcoming investor presentations or SEC filings.
This brief highlights the edition’s top stories. Read the full July 27, 2026 edition → for all stories and analysis — or browse the Rare Disease & Gene Therapy archive.