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pp-243

Injection

Dossier type
US CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Angelman syndrome (genetically confirmed full maternal UBE3A deletion) / Clinical stage: Phase 3 / Modality: Antisense oligonucleotide (ASO) / Mechanism & Target: UBE3A-AS antisense targeting paternal UBE3A allele / Route & form: Subcutaneous injection / Differentiation: Addresses genetic root cause; rare disease with high unmet need; enrollment completed
Manufacturer #39444
Biopharmaceutical company dedicated to rare and ultra-rare genetic diseases. Develops gene therapies and antisense oligonucleotides with multiple Phase 3 programs in advanced clinical stages. Advances transformative treatments targeting severe inherited metabolic disorders.
 

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