AAV miR C9orf 72
Alternative Names: AAV-miR-C9orf-72Latest Information Update: 22 Apr 2024
At a glance
- Originator AviadoBio
- Class Antidementias; Gene therapies; RNA
- Mechanism of Action C9orf72 protein expression modulators; Gene silencing
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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 Amyotrophic lateral sclerosis; Frontotemporal dementia
Most Recent Events
- 18 Apr 2024 Early research in Frontotemporal dementia in United Kingdom (Parenteral) (AviadoBio pipeline, April 2024)
- 02 Jan 2024 Early research in Amyotrophic lateral sclerosis in United Kingdom (Parenteral)
- 23 Feb 2023 AviadoBio in-licenses technology from Neurgain Technologies for novel gene therapy spinal delivery technology