Drug Profile

Gene editing therapy - Evox Therapeutics/ Rett Syndrome Research Trust

Latest Information Update: 24 Apr 2026

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At a glance

Originator Evox Therapeutics
Developer Evox Therapeutics; Rett Syndrome Research Trust
Class Gene therapies
Mechanism of Action Gene transference; Methyl-CpG-binding protein 2 gene replacements

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

27 Feb 2026 Evox Therapeutics enters into a collaboration with Rett Syndrome Research Trust to evaluate ExoEdit^® Technology to enable gene editing in Rett syndrome
27 Feb 2026 Preclinical trials in Rett syndrome in United Kingdom (Parenteral), before February 2026

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