Infrequent
CRISPR Pioneers Jennifer Doudna and Fyodor Urnov Launch Aurora Therapeutics for Custom CRISPR Therapies
Aurora Therapeutics; CRISPR; gene editing; personalized therapies; Jennifer Doudna; Fyodor Urnov; rare diseases; Menlo Ventures
Argenx Appoints Karen Massey as New CEO in Leadership Transition
argenx; Karen Massey; CEO; Tim Van Hauwermeiren; leadership transition; rare diseases; Vyvgart
Shionogi Announces $2.5 Billion Acquisition of ALS Drug Radicava from Tanabe Pharma
Shionogi; Tanabe Pharma; Radicava; ALS; acquisition; $2.5B; rare disease
Spotlight On: Five Key FDA Approvals of 2025
FDA approvals; 2025; orphan drugs; novel therapies; rare diseases
BioMarin Announces $4.8 Billion Acquisition of Amicus Therapeutics for Rare Disease Portfolio Expansion
BioMarin; Amicus Therapeutics; acquisition; $4.8B; Galafold; Pombiliti; Opfolda; rare diseases
Immunome’s desmoid tumor drug varegacestat posts landmark Phase 3 results, setting up a potential challenge to Merck KGaA’s Ogsiveo
Immunome; varegacestat; desmoid tumors; Phase 3 RINGSIDE trial; gamma secretase inhibitor; Merck KGaA; Ogsiveo; progression-free survival; objective response rate; rare tumors; oncology; NDA filing
FDA broadens label for Amgen’s Uplizna as it enters rare disease competition
Uplizna; inebilizumab; Amgen; FDA approval; label expansion; generalized myasthenia gravis; gMG; AChR-positive; MuSK-positive; neuromyelitis optica spectrum disorder; NMOSD; IgG4-related disease; rare disease market; MINT trial; twice-yearly dosing
Cereno Scientific Secures SEK 100m in Directed Share Issue and SEK 350m Loan Financing, Targeting Milestones by Q4 2027
Cereno Scientific; SEK 100 million; Directed share issue; SEK 350 million; Loan financing; Q4 2027 milestones; HDAC inhibitor portfolio; Pulmonary arterial hypertension; Clinical development; Rare cardiovascular diseases
AstraZeneca Onshores Rare Disease Portfolio as Part of $50B US Investment Pledge
AstraZeneca; rare disease portfolio; onshoring; US investment; biomanufacturing; $50 billion pledge; Virginia facility; Maryland investment; R&D expansion
FDA Breakthrough Designations Lead to Priority Reviews, Report Finds
FDA; breakthrough therapy designation; priority review; drug approval; expedited pathway; biotechnology innovation; rare diseases; oncology; CDER; regulatory science