Drug Profile
Wiskott-Aldrich syndrome gene therapy - Genethon
Alternative Names: WAS gene therapy - GenethonLatest Information Update: 15 Feb 2018
Price :
$50
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At a glance
- Originator Genethon
- Developer Childrens Hospital Boston; Genethon
- Class Gene therapies
- Mechanism of Action Gene transference
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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 I/II Wiskott-Aldrich syndrome
Most Recent Events
- 13 Nov 2019 Genethon completes a phase I/II trial for Wiskott-Aldrich syndrome in United Kingdom (IV) (NCT01347242) (ISRCTN46087965)
- 28 Aug 2019 Wiskott-Aldrich syndrome gene therapy is still in phase I/II trial for Wiskott-Aldrich syndrome in France, USA, United Kingdom (IV) (NCT01347346) (NCT01410825)
- 21 Sep 2015 Phase I/II development is ongoing in USA, France and United Kingdom