Drug Profile

Mucopolysaccharidosis IIIC gene therapy - Phoenix Nest

Latest Information Update: 28 Jun 2020

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 University of Manchester
Developer Phoenix Nest; University of Manchester
Class Gene therapies
Mechanism of Action Alpha N acetyl D glucosaminidase replacements; Gene transference

Orphan Drug Status

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

Yes - Mucopolysaccharidosis III
New Molecular Entity No

28 Jun 2020 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-III in USA (Intracerebral)
07 Nov 2018 Phoenix Nest in licensed Mucopolysaccharidosis IIIC gene therapy from University of Manchester
07 Nov 2018 Phoenix Nest announces intention to submit Orphan drug designation application to the US FDA for Mucopolysaccharidosis IIIC

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 (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com