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Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene

Phase 2
Orchard Therapeutics plc (ORTX)

Phase 2

Development Phase

0

Upcoming Catalysts

0

Historical Events

0

Regulatory Approvals

No Catalysts Found

No catalyst events have been recorded for this drug yet.

Competitive Landscape

1 competitor in Mucopolysaccharidosis Type IIIA

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Drug Information

Target Indication

Mucopolysaccharidosis Type IIIA

Clinical Trial

NCT04201405

Last updated: 8/15/2026

Patent & Exclusivity

No patent data available. Pre-approval drugs may not have Orange Book listings.

Drug Science
Mechanism of action & safety profile
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