pp-244 Solution for infusion
Dossier type
US CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Ornithine transcarbamylase (OTC) deficiency / Clinical stage: Phase 3 / Modality: AAV8 gene therapy / Mechanism & Target: OTC gene replacement via AAV8 vector / Route & form: Intravenous injection / Differentiation: Gene therapy addressing genetic root cause; positive efficacy data on ammonia reduction; Phase 3 data continues
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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