Drug Profile
Olenasufligene relduparvovec - LYSOGENE
Alternative Names: AAVrh10-h.SGSH; AAVrh10-hSGSH; LYS-SAF-302; SAF-302; SRP-9002Latest Information Update: 01 Dec 2022
Price :
$50
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At a glance
- Originator LYSOGENE
- Class Gene therapies
- Mechanism of Action Gene transference; N-sulfoglucosamine sulfohydrolase replacements
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Phase II/III Mucopolysaccharidosis III
Most Recent Events
- 29 Nov 2022 Efficacy data from a phase II/III AAVance trial in Mucopolysaccharidosis III released by LYSOGENE
- 29 Nov 2022 Phase-II/III development is ongoing in USA, France, Germany, Netherlands, UK (NCT03612869)
- 07 Jul 2022 Efficacy data from a phase III AAVance trial in Mucopolysaccharidosis III were released by Lysogene