Drug Profile

Research programme: rare genetic diseases gene therapy - Orchard Therapeutics/Fondazione Telethon/Ospedale San Raffaele

Latest Information Update: 03 Oct 2025

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

No development reported Chronic granulomatous disease; Globoid cell leukodystrophy; Metachromatic leukodystrophy; Wiskott-Aldrich syndrome

18 Sep 2025 bluebird bio is now called Genetix Biotherapeutics
24 Jan 2024 Orchard Therapeutics has been acquired by Kyowa Kirin
28 May 2021 No recent reports of development identified for preclinical development in Chronic-granulomatous-disease in Italy

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