Mucopolysaccharidosis IIIC gene therapy - Phoenix Nest
Latest Information Update: 28 Jun 2020
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.
- New Molecular Entity No
Highest Development Phases
- No development reported Mucopolysaccharidosis III
Most Recent Events
- 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