Drug Profile
DO NOT RELEASE - PROFILE HAS TO BE DELETED
Latest Information Update: 25 Feb 2022
Price :
$50
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At a glance
- Originator Fondazione Telethon; GlaxoSmithKline; The San Raffaele Telethon Institute for Gene Therapy
- Developer Orchard Therapeutics
- 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
- No development reported Beta-thalassaemia
Most Recent Events
- 28 Jul 2020 No recent reports of development identified for clinical-Phase-Unknown development in Beta-thalassaemia in United Kingdom (Parenteral)
- 15 Jul 2020 Orchard Therapeutics in-licenses lentiviral stable cell line technology from GlaxoSmithKline
- 29 Apr 2019 Updated efficacy and adverse events data from a proof-of-concept trial in Beta-thalassaemia released by Orchard Therapeutics