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ASO Candidates for SMA

Spinal Muscular Atrophy

Pre-clinicalActive

Key Facts

Indication
Spinal Muscular Atrophy
Phase
Pre-clinical
Status
Active
Company

About Biogen

Biogen stands as a premier biotechnology company focused on developing innovative therapies for complex neurological, immunological, and rare diseases with significant unmet medical needs. With decades of pioneering work in multiple sclerosis and recent expansions into nephrology through the acquisition of Human Immunology Biosciences, the company maintains a diverse pipeline spanning Alzheimer's disease, ALS, lupus, and other challenging conditions. Biogen's commitment to scientific excellence is demonstrated through substantial annual R&D investment of $2 billion and a global team of over 1,300 research professionals driving innovation from discovery to commercialization.

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Other Spinal Muscular Atrophy Drugs

DrugCompanyPhase
GYM329Chugai PharmaceuticalPhase 2
SpinrazaRoyalty PharmaCommercial
EvrysdiRoyalty PharmaCommercial
Spinal Muscular Atrophy ProgramBiogenCommercial
SPINRAZA (nusinersen)Ionis PharmaceuticalsApproved