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Dossier type
US CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Sickle Cell Disease Clinical stage: Phase 3 Modality: Small molecule pyruvate kinase activator Mechanism & Target: PK activator increasing RBC ATP Route & form: Oral Differentiation: First-in-class PK activator for SCD
Manufacturer #39434

Clinical-stage biopharmaceutical company specializing in central nervous system disorders. Develops novel small molecules targeting psychiatric, neurological, and rare CNS conditions. Pursues multiple Phase 2 and Phase 3 programs through global development initiatives.

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