NTLA 2001
Alternative Names: NTLA-2001Latest Information Update: 02 Jul 2024
At a glance
- Originator Intellia Therapeutics
- Developer Intellia Therapeutics; Regeneron Pharmaceuticals
- Class Gene therapies
- Mechanism of Action Gene modulators; Prealbumin expression inhibitors
-
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
- Phase III Transthyretin-related hereditary amyloidosis
Most Recent Events
- 25 Jun 2024 Pharmacodynamics and adverse events data from phase-I trial in Transthyretin-related hereditary amyloidosis released by Intellia Therapeutics
- 09 May 2024 Intellia Therapeutics files an IND application with the FDA in USA for Transthyretin-related hereditary amyloidosis with polyneuropathy, prior to May 2024
- 09 May 2024 Intellia Therapeutics plans to file BLA for the treatment of patients with Transthyretin-related hereditary amyloidosis with polyneuropathy in USA