Drug Profile

Mucopolysaccharidosis IIIC gene therapy - Phoenix Nest

Latest Information Update: 28 Jun 2020

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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

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