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Rare and Neglected Diseases

Scholar Rock: A New Twist on Targeting Growth Factors

Rare and Neglected Diseases

Gene Therapy CDMO Forges Ahead with $120M Series B Financing

Rare and Neglected Diseases

Anima Biotech Heads for the Light with Up-to-$2.4B Takeda Partnership

Rare and Neglected Diseases

From Genomes to Drugs: An Interview with Vertex CSO David Altshuler

Rare and Neglected Diseases

Horizon Therapeutics to Expand Pipeline, R&D with $3B Viela Bio Acquisition

Rare and Neglected Diseases

Horizon Therapeutics Sets Sights on Blockbuster Sales, Broader Pipeline

Rare and Neglected Diseases

Illumina, Emedgene Launch AI-Based Rare Disease Interpretation Partnership

Rare and Neglected Diseases

AstraZeneca to Acquire Alexion for $39B, Adding Rare Disease, Immunology Assets

Rare and Neglected Diseases

AGTC Sets Sights on Late-Stage Retinitis Pigmentosa Gene Therapy Trial

Rare and Neglected Diseases

At Orchard, Moving from Pruning to Planting

Rare and Neglected Diseases

NeuBase Sets a Course for the Clinic

Rare and Neglected Diseases

Trial Triumphs, Takeover Talk Boost Reata Pharmaceuticals

Rare and Neglected Diseases

Gene Therapy Developer Passage Bio Files for $125M IPO

Rare and Neglected Diseases

Sangamo CEO Looks Long-Term toward In Vivo Genome Editing, Regulation

Rare and Neglected Diseases

AGTC Plans Pivotal Trial for XLRP Gene Therapy

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