Drug Profile

Research programme: gene editing therapies - Life Edit Therapeutics

Alternative Names: CFTR gene-editing therapies - Life Edit Therapeutics

Latest Information Update: 28 Apr 2025

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At a glance

Originator Life Edit Therapeutics
Class Antidementias; Antifibrotics; Gene therapies; RNA
Mechanism of Action Cystic fibrosis transmembrane conductance regulator expression modulators; Huntingtin protein expression inhibitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

28 Apr 2025 No recent reports of development identified for research development in Cystic-fibrosis in USA (Parenteral)
25 Apr 2023 Preclinical trials in Huntington's disease in USA (unspecified route)
25 Apr 2023 Pharmacodynamics data from a preclinical trial in Huntington's disease released by Life Edit Therapeutics

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