Infrequent
Spotlight On: AI in Genomics and Multimodal Data Analysis Loosens Orphan Drug R&D Bottlenecks
AI; genomics; multimodal data; orphan drugs; rare diseases; drug discovery; drug repurposing
Surge in Investments Driving Genetic and Pharma Innovations in 2026
genetic medicines; pharma investments; gene therapy; biomanufacturing; venture capital; rare diseases
Rare Disease Vouchers Caught in Political Abyss: Recent Updates on Reauthorization Efforts
Rare Pediatric Disease PRV; Mikaela Naylon Give Kids a Chance Act; Congress reauthorization; FDA priority review voucher; bipartisan support; spending bill
Orphan Drug Trends: A Data-Driven Analysis
orphan drugs; market growth; rare diseases; FDA approvals; genomics; reimbursement challenges
IntraBio’s Levacetylleucine Succeeds in Phase 3 Trial for Ataxia-Telangiectasia, Plans FDA and EMA Approval
IntraBio; levacetylleucine; Ataxia-Telangiectasia; Phase 3; FDA approval; EMA; rare disease
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