Drug Profile
Lenadogene nolparvovec - GenSight Biologics
Alternative Names: GS 010; LUMEVOQ; ND4 gene therapy; rAAV2/2-ND4; RAAV2/2-ND4 vector; rAAV2/2‑ND4 gene therapyLatest Information Update: 15 Mar 2024
Price :
$50
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At a glance
- Originator GenSight Biologics
- Class Eye disorder therapies; Gene therapies
- Mechanism of Action Gene transference; NADH dehydrogenase subunit 4 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 III Leber's hereditary optic atrophy
Most Recent Events
- 12 Mar 2024 GenSight Biologics intends to resume Early Access Program (AAC) in France in Q3 2024
- 25 Jan 2024 GenSight Biologics announces intention to launch lenadogene nolparvovec for Leber's hereditary optic atrophy in USA
- 25 Jan 2024 GenSight Biologics plans to discuss with the UK MHRA to clarify the quickest path towards a regulatory submission