Drug Profile

Research programme: gene-encoded enzyme replacement therapy - SmartPharm Therapeutics

Latest Information Update: 28 Jul 2026

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

28 Jul 2026 No recent reports of development identified for preclinical development in Lysosomal storage diseases in USA (Parenteral, Injection)
29 Jul 2024 Preclinical development is ongoing for Lysosomal storage diseases in USA (Parenteral, Injection) (SmartPharm Therapeutics pipeline, July 2024)
28 Oct 2023 No recent reports of development identified for preclinical development in Lysosomal storage diseases in USA (Parenteral, Injection)

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