Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

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

Next Previous
Search
Drug Profile

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

Latest Information Update: 02 Feb 2024

Price :
$50 *
Note:
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

  • Originator Fondazione Telethon; GlaxoSmithKline; Ospedale San Raffaele
  • Developer Fondazione Telethon; GSK; Ospedale San Raffaele
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • 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

Highest Development Phases

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

Most Recent Events

  • 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
  • 28 May 2021 No recent reports of development identified for preclinical development in Globoid-cell-leukodystrophy in Italy

You need to be a logged in or subscribed to view this content Request demo

If your organization or you do not have a subscription, try one of the following:
  • Contacting your organization’s admin about adding this content to your AdisInsight subscription
  • Buying a PDF version of any individual profile
  • Request a free trial
If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top