Drug Profile
OTL 103
Alternative Names: GSK-2696275; OTL-103; WAS gene therapy - Orchard Therapeutics; WAS transduced autologous CD34+ stem cells - Orchard Therapeutics; WAS transduced CD34+ stem cellsLatest Information Update: 02 Feb 2024
Price :
$50
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At a glance
- Originator Fondazione Telethon; GlaxoSmithKline; Ospedale San Raffaele
- Developer GSK; Orchard Therapeutics; Ospedale San Raffaele
- Class Gene therapies; Haematopoietic stem cells therapies
- Mechanism of Action Cell 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 Wiskott-Aldrich syndrome
Most Recent Events
- 24 Jan 2024 Orchard Therapeutics has been acquired by Kyowa Kirin
- 04 Oct 2023 Ospedale San Raffaele completes a phase I/II trial in Wiskott Aldrich Syndrome in Italy (NCT01515462)
- 10 Jan 2022 Orchard Therapeutics announces intention to submit BLA to the US FDA for Wiskott-Aldrich syndrome in early 2022 (IV)