Rare diseases
While AstraZeneca has discontinued work on four assets—including one in asthma and another in acromegaly—the pharma has also elected to take forward a bispecific antibody that destroys the EGFR protein.
Regeneron hauled in $3.6 billion during the first quarter of 2026, as analysts homed in on a slight Eylea HD miss and key upcoming readouts, including for LAG3 candidate fianlimab in metastatic melanoma.
Chiesi Group is taking KalVista Pharmaceuticals under its wing, paying $1.9 billion for the biotech’s oral therapy Ekterly to treat severe swelling episodes caused by the rare genetic disorder hereditary angioedema.
Staff at Salt Lake City-based techbio company Recursion recently heard from Jenny and Tim Jones about their challenging family history of familial adenomatous polyposis.
The FDA has renewed calls for Amgen’s Tavneos to be pulled from the market, saying it has discovered new evidence that study personnel doctored the results of the drug’s pivotal study in order to make it look effective.
With Phase 3 data in hand, Intellia Therapeutics is seeking approval for its in vivo CRISPR gene editing therapy for hereditary angioedema.
Approved Thursday via the FDA’s Commissioner’s National Priority Voucher program, Otarmeni is the first gene therapy for hearing loss—and the first treatment to target an underlying cause of the condition.
The pivotal study of zilganersen in Alexander disease missed a secondary endpoint, but analysts expect the FDA to approve the asset given the unmet need and overall data.
After receiving the FDA’s greenlight for Hunter syndrome drug Avlayah, Denali Therapeutics CEO Ryan Watts saw the culmination of 20 years of hard work unraveling the mysteries of the blood-brain barrier.
Doubling survival in pancreatic cancer, a long-fought rare disease approval, a massive IPO and ambitious biotech entrepreneurs have BioSpace Senior Editor Annalee Armstrong feeling upbeat about the biotech scene.
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