Research programme: Rett syndrome therapeutics - ProQR Therapeutics/Rett Syndrome Research Trust
Latest Information Update: 11 Jan 2024
At a glance
- Originator ProQR Therapeutics
- Developer ProQR Therapeutics; Rett Syndrome Research Trust
- Class Oligonucleotides
- Mechanism of Action Gene expression modulators
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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 Yes
Highest Development Phases
- Research Rett syndrome
Most Recent Events
- 05 Jan 2024 ProQR Therapeutics enters into research and development collaboration with Rett Syndrome Research Trust (RSRT) to develop RNA-based therapeutics for Rett syndrome
- 05 Jan 2024 Early research in Rett syndrome in Netherlands (Parenteral)
- 05 Jan 2024 Early research in Rett syndrome in USA (Parenteral)