QR 010

Drug Profile

QR 010

Alternative Names: Antisense oligonucleotide targeting the F508delta mutation of CFTR - ProQR Therapeutics; QR 10; QR-010

Latest Information Update: 03 Mar 2017

Price : $50

At a glance

  • Originator ProQR Therapeutics
  • Class Oligoribonucleotides
  • Mechanism of Action Cystic fibrosis transmembrane conductance regulator modulators; RNA interference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Cystic fibrosis
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    Cystic fibrosis
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Cystic fibrosis

Most Recent Events

  • 28 Feb 2017 ProQR Therapeutics plans a phase II trial for cystic fibrosis in 2018
  • 16 Nov 2016 Safety and efficacy data from a phase I trial in Cystic fibrosis released by ProQR Therapeutics
  • 01 Sep 2016 ProQR Therapeutics completes a phase I trial in Cystic fibrosis in USA, Belgium and France (NCT02564354)
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