Drug Profile
Glucocerebrosidase gene therapy - Targeted Genetics
Latest Information Update: 14 May 2019
Price :
$50
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At a glance
- Originator Targeted Genetics
- Class Gene therapies
- Mechanism of Action Gene transference; Glucosylceramidase replacements
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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
- Discontinued Gaucher's disease
Most Recent Events
- 09 May 2019 C3J Therapeutics has merged with AmpliPhi Biosciences Corporation to form Armata Pharmaceuticals
- 25 Oct 2000 Discontinued-I for Gaucher's disease in USA (IV-infusion)
- 03 Mar 1998 Phase-I clinical trials for Gaucher's disease in USA (IV-infusion)