Research programme: gene therapy for Usher Syndrome Type 1 - Institut Pasteur/Sensorion
Alternative Names: USH1G gene therapy; USHER-GTLatest Information Update: 28 May 2023
At a glance
- Originator Institut Pasteur
- Developer Institut Pasteur; Sensorion
- Class Gene therapies
- Mechanism of Action Gene transference; USH1G protein replacements
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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
- No development reported Usher syndromes
Most Recent Events
- 28 May 2023 No recent reports of development identified for preclinical development in Usher syndromes in France (Otic, Injection)