Drug Profile
Research programme: RNA-based therapeutics - ProQR Therapeutics
Alternative Names: Axiomer® EONs; Axiomer® editing oligo nucleotides; QR-411; QRX 021; QRX 1011; QRX 203; QRX 411; QRX 604; QRX 704; QRX 812; QRX 911; QRX-036; QRX-042; QRX-052; Therapeutics for genetic disorders - ProQR; Trident®Latest Information Update: 23 May 2023
Price :
$50
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At a glance
- Originator ProQR Therapeutics
- Class Antidementias; Antifibrotics; Eye disorder therapies; Neuropsychotherapeutics; Oligonucleotides; Skin disorder therapies; Small interfering RNA
- Mechanism of Action RNA interference
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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
- Available For Licensing Yes
Highest Development Phases
- No development reported Alzheimer's disease; Cystic fibrosis; Friedreich's ataxia; Huntington's disease; Mucopolysaccharidosis I; Stargardt disease; Usher syndromes
Most Recent Events
- 31 Dec 2022 ProQR Therapeutics has patent protection for antisense oligonucleotides that are applicable for nucleotide-specific pseudouridylation in South Africa
- 28 Jun 2022 No recent reports of development identified for preclinical development in Usher syndromes in Netherlands (Ophthalmic)
- 28 Oct 2021 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-I in Netherlands