Drug Profile

Research programme: gene therapies - Genethon/Cartesian Therapeutics

Latest Information Update: 28 Feb 2025

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At a glance

Originator Genethon; Selecta Biosciences
Developer Cartesian Therapeutics; Genethon
Class Gene therapies; Hepatoprotectants
Mechanism of Action Immunomodulators

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

Highest Development Phases

No development reported Liver disorders
Discontinued Inborn urea cycle disorders; Muscular dystrophies

Most Recent Events

28 Feb 2025 No recent reports of development identified for preclinical development in Liver-disorders in USA (Parenteral)
23 Aug 2023 Discontinued - Preclinical for Inborn urea cycle disorders in USA (Parenteral)
23 Aug 2023 Discontinued for Muscular dystrophies in USA (Parenteral)

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