Researach programme: gene modulating therapeutics - Scenic Biotech/Genentech
Latest Information Update: 14 Mar 2022
At a glance
- Originator Genentech; Scenic Biotech
- Class Gene therapies
- Mechanism of Action Gene expression modulators
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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
- Research Barth syndrome; Niemann-Pick disease type C
Most Recent Events
- 19 May 2021 Scenic Biotech and Genentech expanded the agreement for the development of gene modifier therapeutics for the treatment of inherited rare diseases
- 02 Feb 2021 Early research in Niemann-Pick disease type C in Netherlands (unspecified route)
- 02 Feb 2021 Early research in Niemann-Pick disease type C in USA (unspecified route)